AnatomyOrganismsDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPsychiatry and PsychologyPhenomena and ProcessesDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaHumanitiesInformation ScienceNamed GroupsHealth CareGeographicals
Amyloid NeuropathiesAmyloid Neuropathies, FamilialPrealbuminAmyloidosisAmyloidDiabetic NeuropathiesAmyloid beta-PeptidesSerum Amyloid A ProteinPeripheral Nervous System DiseasesAmyloid beta-Protein PrecursorPlaque, AmyloidHereditary Sensory and Motor NeuropathyIslet Amyloid PolypeptideCerebral Amyloid AngiopathyHereditary Sensory and Autonomic NeuropathiesOptic Neuropathy, IschemicSerum Amyloid P-ComponentPolyneuropathiesSural NerveAmyloid Precursor Protein SecretasesAlzheimer DiseaseDiflunisalLiver TransplantationComplicityEroticaOligonucleotidesRNAMolecular Sequence DataBase SequenceOligonucleotides, AntisenseAmyloidosis, FamilialHistology, ComparativeCryptococcus gattiiMass SpectrometrySwedenFaculty, MedicalFacultyTandem Mass SpectrometryParaphiliasFaculty, DentalComplement ActivationComplement Pathway, ClassicalComplement C3Syncope, VasovagalTilt-Table TestSyncopeOrthostatic IntoleranceAtrial Natriuretic FactorChemistry, AnalyticHypotension, OrthostaticFlatulenceInstitutionalizationHeartburnDrug-Related Side Effects and Adverse ReactionsDiagnostic Techniques and ProceduresAbbreviations as TopicDigestive System
Amyloid Neuropathies: Disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. Familial, primary (nonfamilial), and secondary forms have been described. Some familial subtypes demonstrate an autosomal dominant pattern of inheritance. Clinical manifestations include sensory loss, mild weakness, autonomic dysfunction, and CARPAL TUNNEL SYNDROME. (Adams et al., Principles of Neurology, 6th ed, p1349)Amyloid Neuropathies, Familial: Inherited disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. The different clinical types based on symptoms correspond to the presence of a variety of mutations in several different proteins including transthyretin (PREALBUMIN); APOLIPOPROTEIN A-I; and GELSOLIN.Prealbumin: A tetrameric protein, molecular weight between 50,000 and 70,000, consisting of 4 equal chains, and migrating on electrophoresis in 3 fractions more mobile than serum albumin. Its concentration ranges from 7 to 33 per cent in the serum, but levels decrease in liver disease.Amyloidosis: A group of sporadic, familial and/or inherited, degenerative, and infectious disease processes, linked by the common theme of abnormal protein folding and deposition of AMYLOID. As the amyloid deposits enlarge they displace normal tissue structures, causing disruption of function. Various signs and symptoms depend on the location and size of the deposits.Amyloid: A fibrous protein complex that consists of proteins folded into a specific cross beta-pleated sheet structure. This fibrillar structure has been found as an alternative folding pattern for a variety of functional proteins. Deposits of amyloid in the form of AMYLOID PLAQUES are associated with a variety of degenerative diseases. The amyloid structure has also been found in a number of functional proteins that are unrelated to disease.Diabetic Neuropathies: Peripheral, autonomic, and cranial nerve disorders that are associated with DIABETES MELLITUS. These conditions usually result from diabetic microvascular injury involving small blood vessels that supply nerves (VASA NERVORUM). Relatively common conditions which may be associated with diabetic neuropathy include third nerve palsy (see OCULOMOTOR NERVE DISEASES); MONONEUROPATHY; mononeuropathy multiplex; diabetic amyotrophy; a painful POLYNEUROPATHY; autonomic neuropathy; and thoracoabdominal neuropathy. (From Adams et al., Principles of Neurology, 6th ed, p1325)Amyloid beta-Peptides: Peptides generated from AMYLOID BETA-PEPTIDES PRECURSOR. An amyloid fibrillar form of these peptides is the major component of amyloid plaques found in individuals with Alzheimer's disease and in aged individuals with trisomy 21 (DOWN SYNDROME). The peptide is found predominantly in the nervous system, but there have been reports of its presence in non-neural tissue.Serum Amyloid A Protein: An ACUTE PHASE REACTION protein present in low concentrations in normal sera, but found at higher concentrations in sera of older persons and in patients with AMYLOIDOSIS. It is the circulating precusor of amyloid A protein, which is found deposited in AA type AMYLOID FIBRILS.Peripheral Nervous System Diseases: Diseases of the peripheral nerves external to the brain and spinal cord, which includes diseases of the nerve roots, ganglia, plexi, autonomic nerves, sensory nerves, and motor nerves.Amyloid beta-Protein Precursor: A single-pass type I membrane protein. It is cleaved by AMYLOID PRECURSOR PROTEIN SECRETASES to produce peptides of varying amino acid lengths. A 39-42 amino acid peptide, AMYLOID BETA-PEPTIDES is a principal component of the extracellular amyloid in SENILE PLAQUES.Plaque, Amyloid: Accumulations of extracellularly deposited AMYLOID FIBRILS within tissues.Hereditary Sensory and Motor Neuropathy: A group of slowly progressive inherited disorders affecting motor and sensory peripheral nerves. Subtypes include HMSNs I-VII. HMSN I and II both refer to CHARCOT-MARIE-TOOTH DISEASE. HMSN III refers to hypertrophic neuropathy of infancy. HMSN IV refers to REFSUM DISEASE. HMSN V refers to a condition marked by a hereditary motor and sensory neuropathy associated with spastic paraplegia (see SPASTIC PARAPLEGIA, HEREDITARY). HMSN VI refers to HMSN associated with an inherited optic atrophy (OPTIC ATROPHIES, HEREDITARY), and HMSN VII refers to HMSN associated with retinitis pigmentosa. (From Adams et al., Principles of Neurology, 6th ed, p1343)Islet Amyloid Polypeptide: A pancreatic beta-cell hormone that is co-secreted with INSULIN. It displays an anorectic effect on nutrient metabolism by inhibiting gastric acid secretion, gastric emptying and postprandial GLUCAGON secretion. Islet amyloid polypeptide can fold into AMYLOID FIBRILS that have been found as a major constituent of pancreatic AMYLOID DEPOSITS.Cerebral Amyloid Angiopathy: A heterogeneous group of sporadic or familial disorders characterized by AMYLOID deposits in the walls of small and medium sized blood vessels of CEREBRAL CORTEX and MENINGES. Clinical features include multiple, small lobar CEREBRAL HEMORRHAGE; cerebral ischemia (BRAIN ISCHEMIA); and CEREBRAL INFARCTION. Cerebral amyloid angiopathy is unrelated to generalized AMYLOIDOSIS. Amyloidogenic peptides in this condition are nearly always the same ones found in ALZHEIMER DISEASE. (from Kumar: Robbins and Cotran: Pathologic Basis of Disease, 7th ed., 2005)Hereditary Sensory and Autonomic Neuropathies: A group of inherited disorders characterized by degeneration of dorsal root and autonomic ganglion cells, and clinically by loss of sensation and autonomic dysfunction. There are five subtypes. Type I features autosomal dominant inheritance and distal sensory involvement. Type II is characterized by autosomal inheritance and distal and proximal sensory loss. Type III is DYSAUTONOMIA, FAMILIAL. Type IV features insensitivity to pain, heat intolerance, and mental deficiency. Type V is characterized by a selective loss of pain with intact light touch and vibratory sensation. (From Joynt, Clinical Neurology, 1995, Ch51, pp142-4)Optic Neuropathy, Ischemic: Ischemic injury to the OPTIC NERVE which usually affects the OPTIC DISK (optic neuropathy, anterior ischemic) and less frequently the retrobulbar portion of the nerve (optic neuropathy, posterior ischemic). The injury results from occlusion of arterial blood supply which may result from TEMPORAL ARTERITIS; ATHEROSCLEROSIS; COLLAGEN DISEASES; EMBOLISM; DIABETES MELLITUS; and other conditions. The disease primarily occurs in the sixth decade or later and presents with the sudden onset of painless and usually severe monocular visual loss. Anterior ischemic optic neuropathy also features optic disk edema with microhemorrhages. The optic disk appears normal in posterior ischemic optic neuropathy. (Glaser, Neuro-Ophthalmology, 2nd ed, p135)Serum Amyloid P-Component: Amyloid P component is a small, non-fibrillar glycoprotein found in normal serum and in all amyloid deposits. It has a pentagonal (pentaxin) structure. It is an acute phase protein, modulates immunologic responses, inhibits ELASTASE, and has been suggested as an indicator of LIVER DISEASE.Polyneuropathies: Diseases of multiple peripheral nerves simultaneously. Polyneuropathies usually are characterized by symmetrical, bilateral distal motor and sensory impairment with a graded increase in severity distally. The pathological processes affecting peripheral nerves include degeneration of the axon, myelin or both. The various forms of polyneuropathy are categorized by the type of nerve affected (e.g., sensory, motor, or autonomic), by the distribution of nerve injury (e.g., distal vs. proximal), by nerve component primarily affected (e.g., demyelinating vs. axonal), by etiology, or by pattern of inheritance.Sural Nerve: A branch of the tibial nerve which supplies sensory innervation to parts of the lower leg and foot.Amyloid Precursor Protein Secretases: Endopeptidases that are specific for AMYLOID PROTEIN PRECURSOR. Three secretase subtypes referred to as alpha, beta, and gamma have been identified based upon the region of amyloid protein precursor they cleave.Alzheimer Disease: A degenerative disease of the BRAIN characterized by the insidious onset of DEMENTIA. Impairment of MEMORY, judgment, attention span, and problem solving skills are followed by severe APRAXIAS and a global loss of cognitive abilities. The condition primarily occurs after age 60, and is marked pathologically by severe cortical atrophy and the triad of SENILE PLAQUES; NEUROFIBRILLARY TANGLES; and NEUROPIL THREADS. (From Adams et al., Principles of Neurology, 6th ed, pp1049-57)Diflunisal: A salicylate derivative and anti-inflammatory analgesic with actions and side effects similar to those of ASPIRIN.Liver Transplantation: The transference of a part of or an entire liver from one human or animal to another.Complicity: Association with or participation in an act that is, or is perceived to be, criminal or immoral. One is complicitous when one promotes or unduly benefits from practices or institutions that are morally or legally suspect.Erotica: Literary or artistic items having an erotic theme. It refers especially to books treating sexual love in a sensuous or voluptuous manner. (Webster, 3d ed)Oligonucleotides: Polymers made up of a few (2-20) nucleotides. In molecular genetics, they refer to a short sequence synthesized to match a region where a mutation is known to occur, and then used as a probe (OLIGONUCLEOTIDE PROBES). (Dorland, 28th ed)RNA: A polynucleotide consisting essentially of chains with a repeating backbone of phosphate and ribose units to which nitrogenous bases are attached. RNA is unique among biological macromolecules in that it can encode genetic information, serve as an abundant structural component of cells, and also possesses catalytic activity. (Rieger et al., Glossary of Genetics: Classical and Molecular, 5th ed)Molecular Sequence Data: Descriptions of specific amino acid, carbohydrate, or nucleotide sequences which have appeared in the published literature and/or are deposited in and maintained by databanks such as GENBANK, European Molecular Biology Laboratory (EMBL), National Biomedical Research Foundation (NBRF), or other sequence repositories.Base Sequence: The sequence of PURINES and PYRIMIDINES in nucleic acids and polynucleotides. It is also called nucleotide sequence.Oligonucleotides, Antisense: Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.Amyloidosis, Familial: Diseases in which there is a familial pattern of AMYLOIDOSIS.Histology, Comparative: The study of the similarities and differences in the structures of homologous tissues across various species.Cryptococcus gattii: A species of the fungus CRYPTOCOCCUS. Its teleomorph is Filobasidiella bacillispora.Mass Spectrometry: An analytical method used in determining the identity of a chemical based on its mass using mass analyzers/mass spectrometers.SwedenFaculty, Medical: The teaching staff and members of the administrative staff having academic rank in a medical school.Faculty: The teaching staff and members of the administrative staff having academic rank in an educational institution.Tandem Mass Spectrometry: A mass spectrometry technique using two (MS/MS) or more mass analyzers. With two in tandem, the precursor ions are mass-selected by a first mass analyzer, and focused into a collision region where they are then fragmented into product ions which are then characterized by a second mass analyzer. A variety of techniques are used to separate the compounds, ionize them, and introduce them to the first mass analyzer. For example, for in GC-MS/MS, GAS CHROMATOGRAPHY-MASS SPECTROMETRY is involved in separating relatively small compounds by GAS CHROMATOGRAPHY prior to injecting them into an ionization chamber for the mass selection.Paraphilias: Disorders that include recurrent, intense sexually arousing fantasies, sexual urges, or behaviors generally involving nonhuman objects, suffering of oneself or partners, or children or other nonconsenting partners. (from DSM-IV, 1994)Faculty, Dental: The teaching staff and members of the administrative staff having academic rank in a dental school.Complement Activation: The sequential activation of serum COMPLEMENT PROTEINS to create the COMPLEMENT MEMBRANE ATTACK COMPLEX. Factors initiating complement activation include ANTIGEN-ANTIBODY COMPLEXES, microbial ANTIGENS, or cell surface POLYSACCHARIDES.Complement Pathway, Classical: Complement activation initiated by the binding of COMPLEMENT C1 to ANTIGEN-ANTIBODY COMPLEXES at the COMPLEMENT C1Q subunit. This leads to the sequential activation of COMPLEMENT C1R and COMPLEMENT C1S subunits. Activated C1s cleaves COMPLEMENT C4 and COMPLEMENT C2 forming the membrane-bound classical C3 CONVERTASE (C4B2A) and the subsequent C5 CONVERTASE (C4B2A3B) leading to cleavage of COMPLEMENT C5 and the assembly of COMPLEMENT MEMBRANE ATTACK COMPLEX.Complement C3: A glycoprotein that is central in both the classical and the alternative pathway of COMPLEMENT ACTIVATION. C3 can be cleaved into COMPLEMENT C3A and COMPLEMENT C3B, spontaneously at low level or by C3 CONVERTASE at high level. The smaller fragment C3a is an ANAPHYLATOXIN and mediator of local inflammatory process. The larger fragment C3b binds with C3 convertase to form C5 convertase.Syncope, Vasovagal: Loss of consciousness due to a reduction in blood pressure that is associated with an increase in vagal tone and peripheral vasodilation.Tilt-Table Test: A standard and widely accepted diagnostic test used to identify patients who have a vasodepressive and/or cardioinhibitory response as a cause of syncope. (From Braunwald, Heart Disease, 7th ed)Syncope: A transient loss of consciousness and postural tone caused by diminished blood flow to the brain (i.e., BRAIN ISCHEMIA). Presyncope refers to the sensation of lightheadedness and loss of strength that precedes a syncopal event or accompanies an incomplete syncope. (From Adams et al., Principles of Neurology, 6th ed, pp367-9)Orthostatic Intolerance: Symptoms of cerebral hypoperfusion or autonomic overaction which develop while the subject is standing, but are relieved on recumbency. Types of this include NEUROCARDIOGENIC SYNCOPE; POSTURAL ORTHOSTATIC TACHYCARDIA SYNDROME; and neurogenic ORTHOSTATIC HYPOTENSION. (From Noseworthy, JH., Neurological Therapeutics Principles and Practice, 2007, p2575-2576)Atrial Natriuretic Factor: A potent natriuretic and vasodilatory peptide or mixture of different-sized low molecular weight PEPTIDES derived from a common precursor and secreted mainly by the HEART ATRIUM. All these peptides share a sequence of about 20 AMINO ACIDS.Chemistry, Analytic: The branch of chemistry dealing with detection (qualitative) and determination (quantitative) of substances. (Grant & Hackh's Chemical Dictionary, 5th ed)Hypotension, Orthostatic: A significant drop in BLOOD PRESSURE after assuming a standing position. Orthostatic hypotension is a finding, and defined as a 20-mm Hg decrease in systolic pressure or a 10-mm Hg decrease in diastolic pressure 3 minutes after the person has risen from supine to standing. Symptoms generally include DIZZINESS, blurred vision, and SYNCOPE.Flatulence: Production or presence of gas in the gastrointestinal tract which may be expelled through the anus.Institutionalization: The caring for individuals in institutions and their adaptation to routines characteristic of the institutional environment, and/or their loss of adaptation to life outside the institution.Heartburn: Substernal pain or burning sensation, usually associated with regurgitation of gastric juice into the esophagus.Drug-Related Side Effects and Adverse Reactions: Disorders that result from the intended use of PHARMACEUTICAL PREPARATIONS. Included in this heading are a broad variety of chemically-induced adverse conditions due to toxicity, DRUG INTERACTIONS, and metabolic effects of pharmaceuticals.Diagnostic Techniques and Procedures: Methods, procedures, and tests performed to diagnose disease, disordered function, or disability.Abbreviations as Topic: Shortened forms of written words or phrases used for brevity.Digestive System: A group of organs stretching from the MOUTH to the ANUS, serving to breakdown foods, assimilate nutrients, and eliminate waste. In humans, the digestive system includes the GASTROINTESTINAL TRACT and the accessory glands (LIVER; BILIARY TRACT; PANCREAS).

Capture of a dimeric intermediate during transthyretin amyloid formation. (1/93)

Point mutations in the human plasma protein transthyretin are associated with the neurological disorder familial amyloidosis with polyneuropathy type 1. The disease is characterized by amyloid fibril deposits causing damage at the site of deposition. Substitution of two amino acids in the hydrophobic core of transthyretin lead to a mutant that was very prone to form amyloid. In addition, this mutant has also been shown to induce a toxic response on a neuroblastoma cell line. Renaturation of the transthyretin mutant at low temperature facilitated the isolation of an amyloid-forming intermediate state having the apparent size of a dimer. Increasing the temperature effectively enhanced the rate of interconversion from a partly denatured protein to mature amyloid. Using circular dichroism the beta-sheet content of the formed mature fibrils was significantly lower than that of the native fold of transthyretin. Morphology studies using electron microscopy also indicated a temperature-dependent transformation from amorphous aggregates toward mature amyloid fibrils. In addition, 1-anilino-8-naphtalenesulfonate fluorescence studies suggested the loss of the thyroxin-binding channel within both the isolated intermediate and the mature fibrils.  (+info)

Deposition of transthyretin in early stages of familial amyloidotic polyneuropathy: evidence for toxicity of nonfibrillar aggregates. (2/93)

Familial amyloidotic polyneuropathy (FAP) is a neurodegenerative disorder characterized by extracellular deposition of transthyretin (TTR) amyloid fibrils, particularly in the peripheral nervous system. No systematic immunohistochemical data exists relating TTR deposition with FAP progression. We assessed nerves from FAP patients in different stages of disease progression (FAP 0 to FAP 3) for TTR deposition by immunohistochemistry, and for the presence of amyloid fibrils by Congo Red staining. The nature of the deposited material was further studied by electron microscopy. We observed that early in FAP (FAP 0), TTR is already deposited in an aggregated nonfibrillar form, negative by Congo Red staining. This suggested that in vivo, preamyloidogenic forms of TTR exist in the nerve, in a stage before fibril formation. Cytotoxicity of nonfibrillar TTR was assessed in nerves of different FAP stages by immunohistochemistry for macrophage colony-stimulating factor. FAP 0 patients already presented increased axonal expression of macrophage colony-stimulating factor that was maintained in all other stages, in sites related to TTR deposition. Toxicity of synthetic TTR fibrils formed in vitro at physiological pH was studied on a Schwannoma cell line by caspase-3 activation assays and showed that early aggregates but not mature fibrils are toxic to cells. Taken together, these results show that nonfibrillar cytotoxic deposits occur in early stages of FAP.  (+info)

Myocardial muscarinic receptor upregulation and normal response to isoproterenol in denervated hearts by familial amyloid polyneuropathy. (3/93)

BACKGROUND: Patients with familial amyloid polyneuropathy, a rare hereditary form of amyloidosis, have progressive autonomic neuropathy. The disease usually does not induce heart failure but is associated with sudden death, conduction disturbances, and an increased risk of complications during anesthesia. Although cardiac sympathetic denervation has been clearly demonstrated, the postsynaptic status of the cardiac autonomic nervous system remains unelucidated. METHODS AND RESULTS: Twenty-one patients were studied (age, 39+/-11 years; normal coronary arteries; left ventricular ejection fraction 68+/-9%). To evaluate the density and affinity constants of myocardial muscarinic receptors, PET with (11)C-MQNB (methylquinuclidinyl benzilate), a specific hydrophilic antagonist, was used. Cardiac beta-receptor functional efficiency was studied by the heart rate (HR) response to intravenous infusion of isoproterenol (5 minutes after 2 mg of atropine, 5, 10, and 15 ng/kg per minute during 5 minutes per step). The mean muscarinic receptor density was higher in patients than in control subjects (B'(max), 35.5+/-8.9 versus 26.1+/-6.7 pmol/mL, P=0.003), without change in receptor affinity. The increase in HR after injection of atropine as well as of MQNB was lower in patients compared with control subjects despite a similar basal HR (DeltaHR after atropine, 11+/-21% versus 62+/-17%; P<0.001), consistent with parasympathetic denervation. Incremental infusion of isoproterenol induced a similar increase in HR in patients and control subjects. CONCLUSIONS: Cardiac autonomic denervation in familial amyloid polyneuropathy results in an upregulation of myocardial muscarinic receptors but without change in cardiac beta-receptor responsiveness to catecholamines.  (+info)

Results of liver transplantation for familial amyloid polyneuropathy type I in Brazil. (4/93)

Familial amyloid polyneuropathy type I (FAP-I) is an inherited amyloidosis secondary to systemic deposition of amyloid fibrils containing mutant transthyretin (TTR) variants. The disease has a progressive clinical course and is usually fatal 10 years after its onset. TTR is mainly produced in hepatocytes, and liver transplantation (LT) has been proposed as an effective treatment for FAP-I. The aim of this study is to evaluate the results of LT for FAP-I in Brazil and analyze prognostic factors associated with survival after surgery. Twenty-four patients (median age, 36 years; range, 25 to 52 years) who underwent LT with the diagnosis of FAP-I were evaluated. Surgery was uneventful in all but six patients who died of complications of primary liver nonfunction (n = 1), cardiogenic shock (n = 1), sepsis (n = 3), and hepatic artery thrombosis (n = 3). Overall 1- and 5-year survival rates were 70% and 58%, respectively. Most patients had stabilization or improvement of symptoms after a median follow-up of 36 months (range, 14 to 82 months). Survivors had a shorter disease duration before LT (median, 6 years; range, 2 to 17 years v 9 years; range, 7 to 12 years; P =.02), greater albumin levels (median, 4 g/dL; range, 3 to 4.7 g/dL v 3.6 g/dL; range, 2.6 to 4.1 g/dL; P =.03), and greater modified body mass index scores (median, 735; range, 502 to 1,432 v 659; range, 411 to 803; P =.04) compared with nonsurvivors. However, only disease duration and albumin levels were independently associated with survival in multivariate analysis. In conclusion, LT is an effective therapy for FAP-I. Mortality after surgery is associated with poor nutritional status and long-standing disease before LT. Thus, LT should be performed as early as possible after the onset of FAP-I symptoms to avoid major disability and improve survival.  (+info)

Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type). (5/93)

Portuguese type familial amyloid polyneuropathy is a dominantly inherited neuropathic amyloidosis caused by a mutant transthyretin (TTR). Because TTR is produced mainly by the liver, liver transplantation (LT) abolishes production of the amyloidogenic variant TTR. To date, the procedure appears to halt the progress of the disease. However, long-term outcome is unknown. The aim of the present study is to evaluate the survival of our initial group of unselected liver transplant recipients with FAP. Seventy patients, 51 transplant recipients and a control group of 19 nontransplantation patients, with disease onset before the age of 55 years were included on the study. Transplant recipients were divided into two categories: (1) early series, with patients followed up for 5 years or longer, and (2) new series, with patients followed up for 1 to 5 years. Nonparametric statistical methods were used. Binary regression analyses were performed by stepwise logistic regression and Cox proportional hazard regression. Survival analysis was performed using Kaplan-Meier analysis, the Cox-Mantel test. Survival analyses and Cox proportional hazard regression analysis were performed from disease onset, not from LT. Significantly decreased survival was noted for transplant recipients with a modified body mass index (mBMI) less than 600 compared with the control group (P < .05). A significant difference in survival also was observed between transplant recipients with an mBMI greater than 600 at the time of LT compared with those with an mBMI less than 600 (P < .02). mBMI and age at LT had a significant impact on survival; whereas late deaths were related to age at LT, early deaths were related to mBMI. The cumulative 10-year survival rate after disease onset was 94% in the new series, with one early death (< 6 months) after LT, compared with a 78% survival rate and eight early deaths in the early series (P = .1).  (+info)

Effect of the intestinal flora on amyloid deposition in a transgenic mouse model of familial amyloidotic polyneuropathy. (6/93)

Familial amyloidotic polyneuropathy (FAP) is a hereditary disease characterized by the systemic accumulation of amyloid fibrils. A mutant transthyretin (TTR) gene is mainly responsible for the disease. However, the variable age of onset and low penetrance might be due to environmental factors, one of which is the intestinal flora. Three types of intestinal flora were introduced into a transgenic (Tg) mouse FAP model, 6.0-hMet30. The CV1 and CV2 group transgenic mice were transferred with the intestinal flora from two different mouse facilities housed under conventional conditions, and the SPF group transgenic mice were kept under specific pathogen free conditions in our facility. All the mice were maintained under controlled temperature, humidity and bacterial conditions. Over a period of 28 months, amyloid was not deposited in the SPF and CV1 groups. In contrast, amyloid was deposited in the esophagus and small intestine of two of the three CV2 mice at 18 months. Many neutrophils infiltrated the lesions. The numbers of tissue neutrophils were higher in the CV2 group than in the SPF and CV1 groups at 18 months. The CV2 flora included fewer gram-positive anaerobic cocci as well as higher proportions of yeasts, staphylococci and enterobacteriaceae compared with the SPF and CV1 flora. These findings suggest that the intestinal flora plays an important role in amyloid deposition.  (+info)

Evidence for early cytotoxic aggregates in transgenic mice for human transthyretin Leu55Pro. (7/93)

Familial amyloidotic polyneuropathy (FAP) is a lethal autosomal dominant disorder characterized by systemic extracellular deposition of transthyretin (TTR) amyloid fibrils. Several groups have generated transgenic mice carrying human TTR Val30Met, the most common mutation in FAP. To study amyloidogenicity and cytotoxicity of different TTRs, we produced transgenic mice expressing human TTR Leu55Pro, one of the most aggressive FAP-related mutations. TTR deposition and presence of amyloid fibrils was investigated and compared to animals carrying the human TTR Val30Met gene kept under the same conditions. Deposition in a C57BL/6J background (TTR-Leu55Pro mice) and in a TTR-null background [TTR-Leu55Pro X TTR-knockout (KO) mice] was compared. Animals in a C57BL/6J background presented early (1 to 3 months) nonfibrillar TTR deposition but amyloid was absent. In a TTR-null background, presence of amyloid fibrils was detected starting at 4 to 8 months with a particular involvement of the gastrointestinal tract and skin. This data suggested that TTR homotetramers are more prone to fibril formation than TTR murine wild-type/human mutant heterotetramers. The nature of the deposited material was further investigated by immunocytochemistry. Both amorphous aggregates and small TTR fibrils were present in TTR-Leu55Pro X TTR-KO transgenics. We observed that these TTR deposits mimic the toxic effect of TTR deposits in FAP: animals with TTR deposition, present approximately twofold increased levels of nitrotyrosine in sites related to deposition. The TTR-Leu55Pro X TTR-KO mice here described are an important tool for the dual purpose of investigating factors involved in amyloidogenesis and in cytotoxicity of deposited TTR.  (+info)

Familial ATTR amyloidosis: microalbuminuria as a predictor of symptomatic disease and clinical nephropathy. (8/93)

BACKGROUND: Portuguese type familial amyloid polyneuropathy (FAP) is a neuropathic amyloidosis caused by a mutant transthyretin (TTR). Varying degrees of renal involvement have been reported. Our aim was to assess the value of microalbuminuria (MA) for predicting clinical neurological disease and overt nephropathy in TTR-related amyloidosis. METHODS: All subjects had the TTR Val30Met mutation, and were recruited between 1993 and 1999. We have prospectively evaluated 22 asymptomatic gene carriers (7 male, 15 female; mean age 41.6+/-9.6 years) and 32 patients with neuropathy (14 male, 18 female; 36.8+/-8.8 years, on average, 33.0+/-9.3 years at the onset of neuropathy). We measured urinary albumin excretion every year, if asymptomatic, or every 6 months if already affected. Kidney biopsies were performed in patients with normal urinary albumin excretion, MA, and overt nephropathy, respectively. RESULTS: In asymptomatic carriers, persistent MA was detected in eight (36%) subjects. The presence of MA in asymptomatic gene carriers, compared with those having normal urinary albumin excretion, conferred a 4.8-fold risk of developing neuropathy, usually within the subsequent 3 years. Once neurological signs appeared, nephropathy, manifested as MA, progressed to overt nephropathy in one-half of subjects. In patients with neuropathy, 24 (75%) had MA during follow-up: evolution towards clinical renal disease occurred in 14 (58%) and renal failure occurred in five (21%), always after a course of MA. Proteinuria or renal failure without prior persistent MA were never observed in the present patient cohort. Histopathological evaluation did not reveal glomerular lesions other than amyloid deposits to explain abnormal urinary albumin excretion. The amount of mesangial and vascular-pole amyloid deposits was correlated with the degree of albuminuria. CONCLUSIONS: Microalbuminuria represents the first stage of clinical TTR amyloid nephropathy and is premonitory of neuropathy. Its presence identifies a subgroup of patients who are more prone to develop overt nephropathy. Screening of MA may be important to assess disease onset and to recommend liver transplantation in individuals at risk.  (+info)

Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, H1 2014- CFDMaster...Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrade's Disease) - Pipeline Review, H1 2014- CFDMaster...
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, H1 2014. Summary. Global Markets Direct s, Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, H1 2014, provides an overview of the indication s therapeutic pipeline. This report provides information on the therapeutic development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease), complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease). Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino de Andrades Disease) - Pipeline Review, Half Year is built using data and information sourced from Global Markets Direct s proprietary databases, Company/University websites, SEC filings, investor ...
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino De Andrades Disease) - Pipeline Review, H1 2014 | Feb 14,...Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis, Corino De Andrade's Disease) - Pipeline Review, H1 2014 | Feb 14,...
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Transthyretin-Related Familial Amyloid Polyneuropathy (TTR-FAP): A Single-Center Experience in Sicily, an Italian Endemic Area ...Transthyretin-Related Familial Amyloid Polyneuropathy (TTR-FAP): A Single-Center Experience in Sicily, an Italian Endemic Area ...
Background: Familial amyloid polyneuropathy related to transthyretin gene (TTR-FAP) is a life-threatening disease transmitted as an autosomal dominant trait. Val30Met mutation accounts for the majority of the patients with large endemic foci especia
Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.govEfficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.gov
Familial Amyloid Polyneuropathy (FAP) is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP.. IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression.. The purpose of this study is to determine if IONIS-TTR Rx can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP patients. Patients will receive either IONIS-TTR Rx or placebo for 65 weeks. ...
Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.govEfficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.gov
Familial Amyloid Polyneuropathy (FAP) is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP.. IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression.. The purpose of this study is to determine if IONIS-TTR Rx can slow or stop the nerve damage caused by TTR deposits. This study will enroll late Stage 1 and early Stage 2 FAP patients. Patients will receive either IONIS-TTR Rx or placebo for 65 weeks. ...
Familial amyloid polyneuropathy (FAP), in an inborn habitat of Hiroshima Prefecture, Japan<...Familial amyloid polyneuropathy (FAP), in an inborn habitat of Hiroshima Prefecture, Japan<...
TY - JOUR. T1 - Familial amyloid polyneuropathy (FAP), in an inborn habitat of Hiroshima Prefecture, Japan. AU - Nitta, K.. AU - Kito, S.. AU - Harada, T.. AU - Sakaki, Y.. AU - Sasaki, H.. PY - 1986/9/1. Y1 - 1986/9/1. UR - http://www.scopus.com/inward/record.url?scp=0022784075&partnerID=8YFLogxK. UR - http://www.scopus.com/inward/citedby.url?scp=0022784075&partnerID=8YFLogxK. M3 - Article. C2 - 3791769. AN - SCOPUS:0022784075. VL - 26. SP - 903. EP - 906. JO - Clinical Neurology. JF - Clinical Neurology. SN - 0009-918X. IS - 9. ER - ...
Familial Amyloid Polyneuropathy Partners Ainlyum and Sanofi End Their AgreementFamilial Amyloid Polyneuropathy Partners Ainlyum and Sanofi End Their Agreement
Read about Alnylam Pharmaceuticals and Sanofi dissolving the partnership they formed to develop two familial amyloid polyneuropathy therapies.
New Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) medicines, recently approved | TheSocialMedworkNew Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) medicines, recently approved | TheSocialMedwork
You can order the latest approved medicines for Neurological Diseases from TheSocialMedwork if the drugs are not available in your country.
Prevalence of hereditary transthyretin amyloid polyneuropathy in idiopathic progressive neuropathy in conurban areas |...Prevalence of hereditary transthyretin amyloid polyneuropathy in idiopathic progressive neuropathy in conurban areas |...
Hereditary transthyretin amyloidosis (ATTR amyloidosis) is a rare, genetically heterogenous, and clinically variable autosomal dominant disease that severely reduces life expectancy. As treatment options grow, a proper diagnostic approach is mandatory especially in non-endemic regions with diverse genetic backgrounds. We examined 102 neuropathy patients at a German neuromuscular centre. Common causes of polyneuropathy were ruled out by medical history and extensive laboratory testing to define a cohort of patients with progressive polyneuropathy classified as idiopathic. Molecular genetic testing of the entire TTR gene was performed, and the detected amyloidogenic and non-amyloidogenic variants were associated with the observed clinical phenotypes and results of prior diagnostic testing. Two of 102 patients tested positive for amyloidogenic mutations (p.Ile127Val and p.Glu81Lys), while a variant of unknown significance, p.Glu26Ser, was found in 10 cases. In both positive cases, previous negative biopsy
The heart in hereditary transthyretin amyloidosis : clinical studies on the impact of amyloid fibril compositionThe heart in hereditary transthyretin amyloidosis : clinical studies on the impact of amyloid fibril composition
Background Hereditary transthyretin amyloid (ATTRm) amyloidosis is a systemic disease mainly affecting the peripheral nervous system and the heart. The disease is inherited in an autosomal dominant manner with a varying penetrance. It is caused by mutations in the transthyretin (TTR) gene. Today more than 100 disease causing mutations are known. The V30M mutation that is endemic in northern Sweden is the best studied and comprises the majority of the reported disease cases in the world. In ATTRm amyloidosis caused by the V30M mutation two distinct sub populations are seen, one with disease onset early in life and a mainly neuropathic disease and the other with late onset disease and both neuropathic disease and a progressive cardiomyopathy. These phenotypical findings have in Swedish patients been tied to differences in amyloid fibril composition. Generally, patients with early onset disease have amyloid fibrils containing only full length transthyretin (type B) whereas patients with late onset ...
Arrhythmia - a pitfall in tests of cardiac autonomic function after liver transplantation for familial amyloidotic...Arrhythmia - a pitfall in tests of cardiac autonomic function after liver transplantation for familial amyloidotic...
Liver transplantation (LT) is a potentially curative treatment for hereditary transthyretin amyloidosis, of which familial amyloid polyneuropathy (FAP) is the most common form in Sweden. This study investigated the long-term development in heart rate variability (HRV) after LT in Swedish FAP patients. HRV was analyzed before LT, and during a first (,40 months) and a second (,40 months) follow-up recording after transplantation, respectively. Power spectrum analysis was performed on 2-min sequences in the supine position and after passive tilt, after careful identification of patients with arrhythmia. Data were obtained from 33 patients, but 18 patients had developed cardiac arrhythmia or were pacemaker-treated (4 before LT and 14 after LT) and three patients had not performed the first follow-up recording. In the remaining 12 patients, HRV decreased between the pretransplant evaluation and the first follow-up, thereafter no significant changes were found. In conclusion, our study showed that the ...
Familial Amyloid Polyneuropathy treatment with Tafamidis - evaluation of one- and two-year treatment in Porto, Portugal |...Familial Amyloid Polyneuropathy treatment with Tafamidis - evaluation of one- and two-year treatment in Porto, Portugal |...
163 patients (92 males), with a mean age of 41.04 ± 11.68 years [26-80] and a mean duration of disease of 29.66 ± 17.48 months [4-90], completed a 12M evaluation. Body mass index remained stable throughout these 12M (3.13 vs. 3.14, p,0.008).. Mean NIS score decreased from baseline to 12M (2.35 vs. 2.34, p,0.694, ns) and Norfolk score improved between baseline and 12M (3.03 vs. 2.74, p,0.000).. Responders (n=112, 68,7%) showed a significant NIS-score decrease between baseline and 12M (2.24 vs. 2.05, p,0.000). Non-responders showed a significant increase across one year (2.56 vs. 2.88, p,0.000). Nonetheless, even in this group there was a Norfolk decreased in the same period (3.27 vs. 3.06, p,0.020).. The group that completed a 24M evaluation consisted of 104 patients (56 males), with a mean age of 40.04 ± 10.14 years [26-76] and a mean duration of disease of 32.03 ± 17.97 months [4-77]. Once again, body mass index remained stable throughout 24M (3.12 vs. 3.13, p,0.414, ns).. Mean NIS score ...
Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin CardiomyopathyPfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy
Vyndaqel (tafamidis) is a new drug in development for the treatment of mild transthyretin familial amyloid polyneuropathy (TTR-FAP) and transthyretin cardiomyopathy (TTR-CM). Vyndaqel information includes news, clinical trial results and side effects.
Curcumin could reduce the monomer of TTR with Tyr114Cys mutation via a | DDDTCurcumin could reduce the monomer of TTR with Tyr114Cys mutation via a | DDDT
Curcumin could reduce the monomer of TTR with Tyr114Cys mutation via autophagy in cell model of familial amyloid polyneuropathy Hui Li,1,* Yu Zhang,1,* Li Cao,1 Ran Xiong,1 Bei Zhang,1 Li Wu,1 Zongbo Zhao,1 Sheng-Di Chen1,2 1Department of Neurology and Institute of Neurology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, 2Key Laboratory of Stem Cell Biology and Laboratory of Neurodegenerative Diseases, Institute of Health Science, Shanghai Institutes of Biological Sciences, Chinese Academy of Science, and Shanghai Jiao Tong University School of Medicine, Shanghai, People’s Republic of China *These authors contributed equally to this work Abstract: Transthyretin (TTR) familial amyloid polyneuropathy (FAP) is an autosomal ­dominant inherited neurodegenerative disorder caused by various mutations in the transthyretin gene. We aimed to identify the mechanisms underlying TTR FAP with Tyr114Cys (Y114C) mutation. Our study showed that TTR
Hereditary Transthyretin amyloidosis | Amyloidosis AllianceHereditary Transthyretin amyloidosis | Amyloidosis Alliance
Their purpose is to prevent the formation of new deposits of amyloidosis by stabilising Transthyretin and blocking its production. The treatments available so far are only able to slow down the progression and even to stop it but not to get rid of the symptoms already present.. a) Liver transplant The purpose of a liver transplant is to remove the main organ producing abnormal TTR protein even if the liver is functioning perfectly well otherwise. A liver transplant is a complicated operation that needs to be performed in a specialized centre.. This treatment has been offered to over 2000 patients worldwide. It has been effective in stopping the progression of the disease in a large majority of cases (70%) treated in their early stages. It is not advised for patients who developed the disease late in life or for carriers of a certain type of mutation. It cannot be performed on patients over 70 years old.. In spite of the transplant, the disease sometimes continues to develop in the nervous system ...
Common Rx Could Slow Neurodegenerative Disease | CARE Pharmacies Cooperative..."Common Rx Could Slow Neurodegenerative Disease" | CARE Pharmacies Cooperative...
NSAID Dolobid inhibited familial amyloid polyneuropathy progression(dailyRx News) Familial amyloid polyneuropathy is a very rare condition. New research shows
Most recent papers with the keyword Cardiac amyloid | Read by QxMDMost recent papers with the keyword Cardiac amyloid | Read by QxMD
Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a progressive, fatal, inherited disorder first identified in Portugal and now recognized in all continents. Over the past decade, thanks to the availability of the genetic test, our knowledge on the range of clinical expressions of this disorder has expanded, including different patterns and progression rates of the neuropathy, as well as aspects of the cardiomyopathy, which can be prominent. In the mean time, new tools are being developed to detect earlier TTR amyloid deposition such as cardiac scintigraphy with technetium-labelled pyrophosphate tracers or small nerve fiber alterations from skin biopsies, or using neurophysiological approaches as well as magnetic resonance neurography (MRN ...
Nerves and neuropathyNerves and neuropathy
Homes.bio.psu.edu • The cells bodies that make these axons sit in the spinal cord and in brainstem and send out axons that contact - Salivary glands in the mouth - Tear glands in the eye - Muscle in the walls of blood vessels - Muscle in the walls of the stomach and What is Neuropathy? • Neuropathy is a general term meaning damage to a nerve • One nerve = mononeuropathy - Example carpal tunnel syndrome • Many nerves = polyneuropathy - Also called peripheral neuropathy Nerve Damage in amyloidosis • Seen in most types - Primary (AL) - Inherited • TTR - also called Familial Amyloid Polyneuropathy • Gelsolin • ILE122 (though not common) - Not typically seen • AA amyloid • Focal amyloid Nerve Damage in Amyloidosis • Can be one nerve - Carpal tunnel syndrome • Can be multiple (but not all) nerves • Can be generalized disorder of nerves - Amyloid polyneuropathy = peripheral Amyloid Polyneuropathy • Axonal, length-dependent, symmetrical, dying- - Axon itself is damaged • ...
Trans-Suppression of Misfolding in an Amyloid Disease -- Hammarström et al. 2001 (2): 18 -- <i>Science</i...Trans-Suppression of Misfolding in an Amyloid Disease -- Hammarström et al. 2001 (2): 18 -- <i>Science</i...
Per Hammarstr�m, Frank Schneider, and Jeffery W. Kelly http://sageke.sciencemag.org/cgi/content/abstract/sageke;2001/2/or18 Abstract: Science 293, 2459-2462 (2001).. The transthyretin (TTR) amyloid diseases, representative of numerous misfolding disorders, are of considerable interest because there are mutations that cause or suppress disease. The Val30>Met30 (V30M) TTR mutation is the most prevalent cause of familial amyloid polyneuropathy in heterozygotes, whereas a Thr119>Met119 (T119M) mutation on the second TTR allele protects V30M carriers from disease. Here, we show that the incorporation of one or more T119M TTR subunits into a predominantly V30M tetramer strongly stabilized the mixed tetramer against dissociation. Dissociation is required for amyloid formation, so these findings provide a molecular explanation for intragenic trans-suppression of amyloidosis. The data also suggest a potential therapeutic strategy, provide insight into tissue-specific deposition and amyloid composition, ...
Journal of Neuromuscular Diseases - Volume 2, issue s2 - Journals - IOS PressJournal of Neuromuscular Diseases - Volume 2, issue s2 - Journals - IOS Press
Authors: Mazzeo, Anna , Russo, Massimo , Di Bella, Gianluca , Minutoli, Fabio , Stancanelli, Claudia , Gentile, Luca , Baldari, Sergio , Carerj, Scipione , Toscano, Antonio , Vita, Giuseppe Article Type: Research Article Abstract: Background: Familial amyloid polyneuropathy related to transthyretin gene (TTR-FAP) is a life-threatening disease transmitted as an autosomal dominant trait. Val30Met mutation accounts for the majority of the patients with large endemic foci especially in Portugal, Sweden and Japan. However, more than one hundred other mutations have been described worldwide. A great phenotypic variability among patients with late- and early-onset has been reported. Objective: To present a detailed report of TTR-FAP patients diagnosed in our tertiary neuromuscular center, in a 20-year period. Methods: Clinical informations were gathered through the database of our center. …Results: The study involved 76 individuals carrying a TTR-FAP mutation. Three phenotypes were identified, each ...
Most recent papers with the keyword Nerve entrapment upper limb | Read by QxMDMost recent papers with the keyword Nerve entrapment upper limb | Read by QxMD
OBJECTIVE: To systematically study peripheral nerve morphology in patients with transthyretin (TTR) amyloidosis and TTR gene mutation carriers using high-resolution ultrasonography (US). METHODS: In this prospective cross-sectional study we took a structured history, performed neurological examination, and measured peripheral nerve cross-sectional areas (CSAs) bilaterally at 28 standard locations using US. Demographic and US findings were compared to controls. RESULTS: Peripheral nerve CSAs were significantly larger in 33 patients with familial amyloid polyneuropathy (FAP) compared to 50 controls, most dramatically at the common entrapment sites (median nerve at the wrist, ulnar nerve at the elbow), and in the proximal nerve segments (median nerve in the upper arm, sciatic nerve in the thigh ...
Gene-Silencing Drugs Finally Show Promise | MIT Technology ReviewGene-Silencing Drugs Finally Show Promise | MIT Technology Review
The disease starts with a feeling of increased clumsiness. Spilling a cup of coffee. Stumbling on the stairs. Having accidents that are easy to dismiss-everyone trips now and then. But it inevitably gets worse. Known as familial amyloid polyneuropathy, or FAP, it can go misdiagnosed for years as patients lose the ability to walk or…
Ionis Pharmaceuticals to host 2020 virtual annual meeting of stockholders | Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals to host 2020 virtual annual meeting of stockholders | Ionis Pharmaceuticals, Inc.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the worlds first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardiovascular, infectious, and pulmonary diseases.. To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.. ...
Ionis Pharmaceuticals to present at upcoming virtual investor conferences | Ionis Pharmaceuticals, Inc.Ionis Pharmaceuticals to present at upcoming virtual investor conferences | Ionis Pharmaceuticals, Inc.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the worlds first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardiovascular, infectious, and pulmonary diseases.. To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.. ...
FDA Staff Pans Drug for Rare Nerve Disease | Medpage TodayFDA Staff Pans Drug for Rare Nerve Disease | Medpage Today
WASHINGTON -- An investigational drug to treat familial amyloid polyneuropathy, a rare neurodegenerative disease, should not be approved, according to FDA reviewers.
All News | ALZFORUMAll News | ALZFORUM
By stopping familial amyloid polyneuropathy in its tracks, a repurposed anti-inflammatory medication supports the idea that artificial chaperones can prevent protein aggregation.. ...
Alnylam Notches a Game-Changing RNAi Win -- The Motley FoolAlnylam Notches a Game-Changing RNAi Win -- The Motley Fool
Alnylams patisiran may soon become the standard of care in treating hereditary transthyretin amyloidosis in patients with widespread neuropathy.. In phase 3 trials, patisiran significantly reduced the progression of neuropathy and it improved patients quality of life. Specifically, 225 patients were enrolled in a study comparing patisiran to placebo. Patients were treated for 18 months and patients receiving patisiran experienced a highly significant reduction in neuropathy versus placebo, as measured by a composite neuropathy score, with a p-value of 0.00001. Patients also experienced an improvement in the quality of life as measured by a questionnaire.. Despite worry over safety risks associated with interfering with RNA, patisirans safety profile doesnt appear to raise a lot of eyebrows. The rate of adverse events in the patisiran and placebo arms was similar (96.6% and 97.4%, respectively), as was the rate of serious adverse events (36.5% and 40.3%, respectively).. ...
Polyneuropathies | Harvard Catalyst Profiles | Harvard CatalystPolyneuropathies | Harvard Catalyst Profiles | Harvard Catalyst
Adams D, Gonzalez-Duarte A, ORiordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 07 05; 379(1):11-21 ...
Transthyretin-Derived (ATTR) Amyloidotic Cardiomyopathy After Receiving a Domino Liver Allograft | CirculationTransthyretin-Derived (ATTR) Amyloidotic Cardiomyopathy After Receiving a Domino Liver Allograft | Circulation
A 67-year-old man presented with neuropathy. Five years earlier he had undergone liver transplantation because of cirrhosis caused by hemochromatosis with multiple foci of hepatocellular carcinoma. The donor liver was procured from a patient with transthyretin-derived (ATTR) amyloidosis caused by a TTR mutation (p.Val71Ala). A fat-biopsy showed Congo red-positive amyloid deposits (Figure 1A) with green birefringence (Figure 1B). Two years after presentation (7 years after transplantation) bone scintigraphy with 99mTc-hydroxyethylene diphosphonate showed uptake in several soft tissues as well as the heart (Figure 2).1 Echocardiography at that time still showed normal left ventricular end-diastolic volume (74 mL/m2), normal left ventricular function (ejection fraction, 56%; global longitudinal systolic strain, 17.4%), normal wall thickness (interventricular septum, 10 mm; posterior wall, 10 mm; and right ventricular wall, 5 mm), normal diastolic function (septal e′ 10.4 cm/s and lateral e′ ...
Akcea Therapeutics Genetic Disease Treatment Gets FDA Clearance - Drug Discovery and DevelopmentAkcea Therapeutics Genetic Disease Treatment Gets FDA Clearance - Drug Discovery and Development
The FDA approved Akcea Therapeutics Tegsedi (inotersen) for the treatment of polyneuropathy caused by hereditary transthyretin (hATTR)-mediated amyloidosis in adults. The self-administered RNA-targeting therapeutic was approved by European agencies three months ago.. Tegsedis approval has come shorty after the FDA clearance of Alnylam Pharmaceuticals Onpattro (patisiran) for the treatment of adults with polyneuropathy due to hATTR, marking the first drug authorized for this indication in the U.S.. While Akcea has currently priced Tegsedi at a similar level to Onpattro (around $345,000 to $450,000 a year), Akcea CEO Paul Soteropoulos commented that the company is considering strategies to increase affordability. She noted that "its self-administration gives the flexibility to treat at a time that works for [patients], which could change the way this progressive and debilitating disease is treated and managed." Onpattro is administered via infusion at a clinic once every three weeks. Despite ...
Attribute::Handlers - search.cpan.orgAttribute::Handlers - search.cpan.org
my MyClass $slr :Good :Bad(1**1-1) :Omni(-vorous); MyClass::Good:ATTR(SCALAR)( MyClass, # class LEXICAL, # no typeglob \$slr, # referent Good, # attr name undef # no attr data CHECK, # compiler phase ); MyClass::Bad:ATTR(SCALAR)( MyClass, # class LEXICAL, # no typeglob \$slr, # referent Bad, # attr name 0 # evald attr data CHECK, # compiler phase ); MyClass::Omni:ATTR(SCALAR)( MyClass, # class LEXICAL, # no typeglob \$slr, # referent Omni, # attr name -vorous # evald attr data CHECK, # compiler phase ); # sub fn :Ugly(sister) :Omni(po,tent()) {...} MyClass::UGLY:ATTR(CODE)( SomeOtherClass, # class \*SomeOtherClass::fn, # typeglob \&SomeOtherClass::fn, # referent Ugly, # attr name sister # evald attr data CHECK, # compiler phase ); MyClass::Omni:ATTR(CODE)( SomeOtherClass, # class \*SomeOtherClass::fn, # typeglob \&SomeOtherClass::fn, # referent Omni, # attr name [po,acle] # evald attr data CHECK, # compiler phase ); # my @arr :Good ...
Contest Founders Enthusiasm Has Domino Effect - tribunedigital-sunsentinelContest Founder's Enthusiasm Has Domino Effect - tribunedigital-sunsentinel
As usual, Joel Campbell will be playing dominoes with his friends this weekend.But this time, hes expecting about 500 other players to show up.Campbell is the brains behind the biggest dominoes
TIDNINGSARKIVET.SE: DOMINO UNICORN, SPELTIDNINGSARKIVET.SE: DOMINO UNICORN, SPEL
DOMINO UNICORN, SPEL i Tidningsarkivet. Ett digitalt arkiv för svenska tidningar och tidskrifter. Här finns bland annat omslag och innehållstexter för DOMINO UNICORN, SPEL.
Too Many Questions: April 2016Too Many Questions: April 2016
8. If any two sequences match, those sequences are attracted to each other and their union spawns a new sequence, which is not a perfect replica of its parents; one end of the sequence will always gain or lose one domino. If last domino in the new sequence is connected to the next by a number which is less than half the domino sets maximum number the last domino is lost from the sequence, breaks of and becomes part of the pool, if more than half it gains. If gained it gains it, where a parent has a matching end domino, from one of the parents. The new sequence then becomes part of the pool of sequences and is attracted to any other matching sequences(or loose dominoes if it was spawned of parents with unmatched gain potential). Repeat this process until all attraction couplings are complete ...
Domino Data Lab | SASDomino Data Lab | SAS
SAS and Domino join forces to streamline SAS users ability to run data science workloads in the cloud. Using SAS for Containers on Domino, users such as data scientists, statisticians and business analysts can create SAS programs, develop SAS models, and publish applications.
Mewtwo ReturnsMewtwo Returns
Domino picks up one of the baby Nidoqueen and admires it. Luna telling her thats dangerous, and the mother Nidoqueen leaps at Domino along with Ryhorn. Domino simply throws her rose and it shocks the two severely, and catches them. She tells how ruthlessly she steals Pok mon. Domino finds Jesse and James and gloats how well she works, Jesse being jealous. Again the negotiations are on with Mewtwo and Giovanni. Everyone decides to help Mewtwo if possible. Mewtwo allows them to levitate and they go for the army. Giovanni tells Domino to evac, but is too late, as well as Mewtwo. Once again Giovanni appears and asks again for Mewtwo to join or every Pok mon will suffer more. The two machines from before appear and create a energy sphere. Giovanni gives him a choice, submit, or everyoen gets it. Mewtwo can do a thing about. Domino shows theyre not kidding by blasting both Pikachus with her rose. (whats in it?) Mewtwo goes forward, the clones telling him not to, still he does. Mewtwo enters the ...
Biomod/2012/HKBU/BU Magician - OpenWetWareBiomod/2012/HKBU/BU Magician - OpenWetWare
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Setting a Fall Table with Domino by Lenox Gradiance Collection for AnthropologieSetting a Fall Table with Domino by Lenox Gradiance Collection for Anthropologie
Inspired by an autumnal palette, our Domino by Lenox Gradiance Collection for Anthropologie is our newest go-to for elevating our entertaining game at every meal. Get the scoop on the stunning collection here.
Fats Domino - Troubles of My Own (2012) - бесплатно слушать онлайн 21 трек с альбомаFats Domino - Troubles of My Own (2012) - бесплатно слушать онлайн 21 трек с альбома
Слушайте музыку с альбома Troubles of My Own от Fats Domino бесплатно онлайн или сохраняйте на компьютер. А также более 15 000 000 mp3 треков в свободном доступе для бесплатного прослушивания.
anova - creating contrast matrix (limma) for two factorial in R - Cross Validatedanova - creating contrast matrix (limma) for two factorial in R - Cross Validated
design g1.s g1.c g2.s g2.c g3.s g3.c g4.s g4.c g5.s g5.c 1 1 0 0 0 0 0 0 0 0 0 2 1 0 0 0 0 0 0 0 0 0 3 1 0 0 0 0 0 0 0 0 0 4 0 1 0 0 0 0 0 0 0 0 5 0 1 0 0 0 0 0 0 0 0 6 0 1 0 0 0 0 0 0 0 0 7 0 0 1 0 0 0 0 0 0 0 8 0 0 1 0 0 0 0 0 0 0 9 0 0 1 0 0 0 0 0 0 0 10 0 0 0 1 0 0 0 0 0 0 11 0 0 0 1 0 0 0 0 0 0 12 0 0 0 1 0 0 0 0 0 0 13 0 0 0 0 1 0 0 0 0 0 14 0 0 0 0 1 0 0 0 0 0 15 0 0 0 0 1 0 0 0 0 0 16 0 0 0 0 0 1 0 0 0 0 17 0 0 0 0 0 1 0 0 0 0 18 0 0 0 0 0 1 0 0 0 0 19 0 0 0 0 0 0 1 0 0 0 20 0 0 0 0 0 0 1 0 0 0 21 0 0 0 0 0 0 1 0 0 0 22 0 0 0 0 0 0 0 1 0 0 23 0 0 0 0 0 0 0 1 0 0 24 0 0 0 0 0 0 0 1 0 0 25 0 0 0 0 0 0 0 0 1 0 26 0 0 0 0 0 0 0 0 1 0 27 0 0 0 0 0 0 0 0 1 0 28 0 0 0 0 0 0 0 0 0 1 29 0 0 0 0 0 0 0 0 0 1 30 0 0 0 0 0 0 0 0 0 1 attr(,"assign") [1] 1 1 1 1 1 1 1 1 1 1 attr(,"contrasts") attr(,"contrasts")$factors [1] "contr.treatment ...
When Fats Domino and The Singing Dogs Ruled the Pop Charts | PopBopRocktilUDropWhen Fats Domino and The Singing Dogs Ruled the Pop Charts | PopBopRocktilUDrop
The Early Global Million Selling Records In the beginning - Gold Record awards in the United States, was determined by record companies tracking their own sales. The first Gold Record was Glenn Millers 1942 release of Chattanooga Choo Choo. Elvis would be recognized by RCA for his 1956 smash hit of Dont Be Cruel. Recording…
Historie verzí stránky „Tips serta Strategi Menang Pada Domino Online - WAdminHistorie verzí stránky „Tips serta Strategi Menang Pada Domino Online" - WAdmin
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Addgene: 251 pTol2DestAddgene: 251 pTol2Dest
Plasmid 251 pTol2Dest from Dr. Nathan Lawsons lab contains the insert attR1/attR2 cassette and is published in Dev Dyn. 2007 Nov . 236(11):3077-87. This plasmid is available through Addgene.
Investigating the Mechanisms of Transthyretin Amyloid Aggregation - Fight Aging!Investigating the Mechanisms of Transthyretin Amyloid Aggregation - Fight Aging!
The tetrameric thyroxine transport protein transthyretin (TTR) forms amyloid fibrils upon dissociation and monomer unfolding. The aggregation of TTR causes life-threatening transthyretin amyloidosis (ATTR) associated with three conditions traditionally known as senile systemic amyloidosis, familial amyloidotic polyneuropathy, and familial amyloidotic cardiomyopathy. Senile systemic amyloidosis is a late onset disease in which Tafamidis, a TTR tetramer stabilizer, has been recently approved in Europe; it delays progression of the disease. Several other therapeutics are currently in clinical trials, including other tetramer stabilizers such as diflunisal and RNAi therapies that cause a decrease in the production of TTR protein. Additional approaches are needed to prevent ATTR, and here we explore the use of peptide inhibitors that block aggregation of TTR. Several models of the TTR amyloid spine have been proposed, but the aggregation-prone segments of the protein remain uncertain. Based on the ...
RNA interference-based therapy for transthyretin amyloidosisRNA interference-based therapy for transthyretin amyloidosis
Transthyretin amyloidosis is caused by deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and heart. A paper recently published in the New England Journal of Medicine reports the safety and efficacy of a potent antitransthyretin small interfering RNA (RNAi) encapsulated in lipid nanoparticles and injected in patients with transthyretin amyloidosis. The RNAi resulted in sustained reduction of transthyretin levels. This study establishes a proof of concept for RNAi therapy targeting messenger RNA transcribed from a disease-causing gene.. ...
Extensive AL Amyloidosis Presenting with Recurrent Liver Hemorrhage and Hemoperitoneum: Case Report and Literature Review |...Extensive AL Amyloidosis Presenting with Recurrent Liver Hemorrhage and Hemoperitoneum: Case Report and Literature Review |...
1. Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med 2003; 349(6): 583-596. 2. Loss M, Ng WS, Karim RZ et al. Hereditary lysozyme amyloidosis: spontaneous hepatic rupture (15 years apart) in mother and daughter. role of emergency liver transplantation. Liver Transpl 2006; 12(7): 1152-1155. 3. Michowska M, Boj E, Wrzołkowa T et al. A First Case of Liver Rupture In Transthyretin (TTR) Familial Amyloid Polyneuropathy. Exp Clin Hep 2005; 1(2): 109-112. 4. Gertz MA, Kyle RA. Hepatic amyloidosis: clinical appraisal in 77 patients. Hepatology 1997; 25(1): 118-121. 5. Comenzo RL, Zhang Y, Martinez C et al. The tropism of organ involvement in primary systemic amyloidosis: contributions of Ig V(L) germ line gene use and clonal plasma cell burden. Blood 2001; 98(3): 714-720. 6. Solomon A, Macy SD, Wooliver C et al. Splenic plasma cells can serve as a source of amyloidogenic light chains. Blood 2009; 113(7): 1501-1503. 7. Park MA, Mueller PS, Kyle RA et al. Primary (AL) hepatic ...
Petersens hernia after living donor liver transplantation | Surgical Case Reports | Full TextPetersen's hernia after living donor liver transplantation | Surgical Case Reports | Full Text
The 44-year-old man in this study was presented with sudden-onset, persistent epigastralgia and had undergone living donor living transplantation (LDLT) for familial amyloid polyneuropathy at 42 years of age, with the left hepatic lobe graft donated by his wife. During LT, biliary reconstruction was performed by hepaticojejunostomy with a Roux limb via the antecolic route as the common bile duct was removed for the sake of the following domino LT. The peritoneal defect related to Roux-en-Y anastomosis was primarily closed with several 4-0 silk interrupted sutures. Although he had experienced repeated episodes of small bowel obstruction, which had all recovered fully following conservative management, at 5, 9, and 14 months post-transplantation, continuous epigastralgia and repeated vomiting for 7 h during the present admission prompted clinical suspicion of bowel strangulation. Abdominal guarding and rigidity in the epigastric region were noted on examination. The body temperature was 37.1 °C. ...
Iodine-123 Metaiodobenzylguanidine Scintigraphic Assessment of Myocardial Sympathetic Innervation in Patients With Familial...Iodine-123 Metaiodobenzylguanidine Scintigraphic Assessment of Myocardial Sympathetic Innervation in Patients With Familial...
In the present study, we found complete defects on MIBG myocardial scans in 8 of 12 patients and limited uptake in the remaining 4 in association with severe systemic autonomic dysfunction. The incidence and magnitude of myocardial accumulation of MIBG were independent of clinical findings, including neurologic disabilities, duration of the illness, extent of endomyocardial amyloid deposition, ECG QRS voltage and ventricular wall thickness. These findings strongly suggest that cardiac adrenergic denervation due to autonomic nervous degeneration ([28]) accounts for alterations in I-123 MIBG myocardial imaging in patients with familial amyloid polyneuropathy. The presence of small localized concentrations of MIBG in the LV anterior wall in some patients indicates that myocardial sympathetic innervation is not equally impaired in this disease.. As we have previously reported ([34]), Tc-99m PYP scintigraphy may have the potential to detect early myocardial amyloid infiltration in patients with ...
Regenerative Biology and Medicine | Leaders in Pharmaceutical Business Intelligence (LPBI) GroupRegenerative Biology and Medicine | Leaders in Pharmaceutical Business Intelligence (LPBI) Group
Familial transthyretin amyloid polyneuropathy. Curator: Larry H. Bernstein, MD, FCAP. LPBI. First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded TTR Monomers and Oligomers in FAP Patients. We reported data from our ongoing Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic polyneuropathy (FAP). Alnylam scientists and collaborators from The Scripps Research Institute and Misfolding Diagnostics, Inc. were able to measure the effects of patisiran on pathogenic, misfolded TTR monomers and oligomers in FAP patients. Results showed a rapid and sustained reduction in serum non-native conformations of TTR (NNTTR) of approximately 90%. Since NNTTR is pathogenic in ATTR amyloidosis and the level of NNTTR reduction correlated with total TTR knockdown, these results provide direct mechanistic evidence supporting the therapeutic ...
Wild-type transthyretin amyloid - WikipediaWild-type transthyretin amyloid - Wikipedia
Wild-type transthyretin amyloid (WTTA), also known as senile systemic amyloidosis (SSA) and abbreviated as ATTR, is a disease that typically affects the heart and tendons of elderly people. It is caused by accumulation of a wild-type (that is to say a normal) protein called transthyretin. This is in contrast to a related condition called transthyretin-related hereditary amyloidosis where a genetically mutated transthyretin protein tends to deposit at a much earlier age than in WTTA, due to abnormal conformation and bioprocessing. It belongs to a group of diseases called amyloidosis, chronic progressive conditions linked to abnormal deposition of normal or abnormal proteins, because these proteins are misshapen and cannot be properly degraded and eliminated by the cell metabolism. Wild-type transthyretin amyloid accumulates mainly in the heart, where it causes stiffness and often thickening of its walls, leading consequently to shortness of breath and intolerance to exercise, called diastolic ...
Pathogenesis of transthyretin amyloidosis<...Pathogenesis of transthyretin amyloidosis<...
TY - JOUR. T1 - Pathogenesis of transthyretin amyloidosis. AU - Benson, Merrill. PY - 2012/6. Y1 - 2012/6. N2 - Current dogma for transthyretin (TTR) pathogenesis is that mutations in TTR alter its structure such that the tetramer becomes unstable and prone to release of monomer which then becomes the putative building block of the fibril. This hypothesis is supported by thermodynamic data showing decreased stability of mutant TTR tetrameric proteins and accelerated fibril formation under acidic conditions in vitro. There are, however, a number of questions that are not readily answered by this simplistic model of a very complex disease. Worrisome questions still to be answered include: 1. If the monomer is the precursor of the fibril, why do fibril deposits contain large amounts of wild-type TTR and not just variant 2. If destabilized tetramers can form fibrils in vitro, why do we consistently find partial proteolysis of fibril subunit proteins If enzymatic proteolysis is a required step in ...
Distal bicep tendon rip tied to transthyretin amyloidosisDistal bicep tendon rip tied to transthyretin amyloidosis
(HealthDay)-Rupture of the distal biceps tendon (RBT) in a patient with heart failure with preserved ejection fraction should raise suspicion for wild-type transthyretin amyloidosis (ATTRwt), according to a research letter ...
Growth of ATTR Market (Transthyretin Amyloidosis Industry) to be Driven by Upcoming Major Drugs | Bismarck News UpdatesGrowth of ATTR Market (Transthyretin Amyloidosis Industry) to be Driven by Upcoming Major Drugs | Bismarck News Updates
Methods to diagnose ATTR include tissue biopsy, genetic testing and imaging studies of the heart.. Currently, there are no higher efficacy ATTR drugs available in the market, but they are expected to hit the market by 2018. The potential ATTR drug candidates include Patisiran and IONIS-TTRrx. For now, off-label drugs and therapies are employed to counter the progression of ATTR.. Request Sample of this Report at: http://www.orbisresearch.com/contacts/request-sample/366384. The global ATTR market is expected to experience robust growth post the launch of ATTR therapeutic drugs, primarily due to increasing African-American population, increasing health care expenditure and accelerating economic growth. However, the growth of this budding market is hindered by the stringent regulations, high cost of ATTR drugs, misdiagnosis of ATTR disorder and limitation of clinical trials.. The major trends, growth drivers as well as issues being faced by the market are discussed in detail in this report.. The ...
Amyloid Polyneuropathy, Familial - Medical Dictionary online-medical-dictionary.orgAmyloid Polyneuropathy, Familial - Medical Dictionary online-medical-dictionary.org
Inherited disorders of the Peripheral Nervous System associated with the deposition of Amyloid in nerve Tissue. The different clinical types based on symptoms correspond to the presence of a variety of Mutations in several different Proteins including Transthyretin (Prealbumin); Apolipoprotein A-I; and Gelsolin ...
Liver Transplantation in HIV and HCV Coinfected PatientsLiver Transplantation in HIV and HCV Coinfected Patients
RESULTS. 3 patients received a domino graft (from a donor transplanted for familial amyloidotic polyneuropathy); 2 a living related donor graft and 2 a cdaver graft. 5 of the 7 are alive, and as the presenter said 4 are "alive and well", but post transplant experience can have complications which are described below. The average followup time for these patients is 12.8 months (4-30 months). The longest a patient is alive who is doing well is 30 months. In general transplant experience is that patients with hepatitis B have better outcomes than patients with hepatitis C. 3 patients are doing relatively well. The study presenter said 4 of the 7 patients have shown dramatic improvement. 1 patient is in good condition at month 30 and is HCV negative. a second patient has F1 fibrosis at month 12 of followup with low or undetectable HIV RNA and in good condition. A third patient is in good condition at month 18 of followup. Three patients are not doing well. Another patient is alive with F3 fibrosis ...
Amyloid Fibril Composition as a Predictor of Development of Cardiomyopathy After Liver Transplantation for Hereditary...Amyloid Fibril Composition as a Predictor of Development of Cardiomyopathy After Liver Transplantation for Hereditary...
Background. Liver transplantation (LTx) is an accepted treatment for hereditary transthyretin (TTR) amyloidosis (ATTR). However, unforeseen heart complications, especially a rapid development of cardiomyopathy after LTx has affected mortality and morbidity. Recently, a relationship between ATTR-fibril composition and cardiomyopathy has been noted. The aim of this study was to investigate whether development of cardiomyopathy and heart failure in LTx ATTR amyloid patients is related to amyloid fibril composition.. Methods. Twenty-four patients with hereditary ATTR amyloidosis who had undergone LTx and have had their amyloid fibril type tested were available for the study. They had been examined by echocardiography including tissue Doppler and speckle tracking echocardiography before and after LTx. Patients were divided into two groups according to fibril composition, 10 patients with type A fibrils (a mixture of truncated and full-length TTR) and 14 patients with type B fibrils (full-length TTR ...
Native T1 mapping in transthyretin amyloidosis. - PubMed - NCBINative T1 mapping in transthyretin amyloidosis. - PubMed - NCBI
JACC Cardiovasc Imaging. 2014 Feb;7(2):157-65. doi: 10.1016/j.jcmg.2013.10.008. Epub 2014 Jan 8. Comment; Comparative Study; Research Support, Non-U.S. Govt
KAKEN - Research Projects | Development of drugs targeting secretory inhibition of transthyretin for familial amyloidotic...KAKEN - Research Projects | Development of drugs targeting secretory inhibition of transthyretin for familial amyloidotic...
Principal Investigator：SATO Takashi, Project Period (FY)：2012-04-01 - 2014-03-31, Research Category：Grant-in-Aid for Young Scientists (B), Research Field：Biological pharmacy
Cardiologic Phenotypes and Natural History of FAC | Springer for Research & DevelopmentCardiologic Phenotypes and Natural History of FAC | Springer for Research & Development
Fifty years ago, the focus on the Val30Met type of the disease, in which neurologic manifestations predominate, led to the widespread notion that hereditary transthyretin-related amyloidosis (ATTR) was essentially a neurologic disease. It is now clear that ATTR is extremely heterogeneous on both genotypic and phenotypic grounds. The clinical spectrum of the disease ranges from an almost exclusive neurologic involvement to strictly cardiac manifestations. This heterogeneity is linked to several factors including specific transthyretin mutations, geographic distribution and endemic vs. non-endemic aggregation type. The existence of exclusively or predominantly cardiac phenotypes makes the recognition of the disease very challenging since it can mimic other more common causes of left ventricular "hypertrophy". Assessment of such patients should include an active search for possible red flags that can indicate the correct final diagnosis. More in general the clinician must be aware that: ...
Clinical Research | Amyloidosis CenterClinical Research | Amyloidosis Center
B3461028: The Tafamidis Study - A Multicenter, International, Phase 3, Double-Blind, Placebo-Controlled, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826) 20 mg or 80 mg in Comparison to Placebo in Subjects Diagnosed With Transthyretin Cardiomyopathy (TTR-CM) ...
Symptoms of Toxic polyneuropathy -- Perhexiline - RightDiagnosis.comSymptoms of Toxic polyneuropathy -- Perhexiline - RightDiagnosis.com
Symptoms of Toxic polyneuropathy - Perhexiline including 11 medical symptoms and signs of Toxic polyneuropathy - Perhexiline, alternative diagnoses, misdiagnosis, and correct diagnosis for Toxic polyneuropathy - Perhexiline signs or Toxic polyneuropathy - Perhexiline symptoms.
Double 6 Dominoes Set - Black Back - White Croco Case - Luxury Domino Sets - Premium Quality Dominoes - GammonVillage Store USADouble 6 Dominoes Set - Black Back - White Croco Case - Luxury Domino Sets - Premium Quality Dominoes - GammonVillage Store USA
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IBM Enterprise Integrator for Domino - Details - SwedenIBM Enterprise Integrator for Domino - Details - Sweden
IBM Enterprise Integrator for Domino (IBM Lotus Integrator for Domino or LEI) connects Domino applications to third-party data to improve business processes
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DCE 1.1: Authentication and Security Services - sec rgy attr test and updateDCE 1.1: Authentication and Security Services - sec rgy attr test and update
The sec_rgy_attr_test_and_update() routine updates an attribute only if the set of control attributes specified in the test_attrs match attributes that already exist for the object. This update is an atomic operation: if any of the control attributes do not match existing attributes, none of the updates are performed, and if an update should be performed, but the write cannot occur for whatever reason to any member of the update_attrs array, all updates are aborted. The attribute causing the update to fail is identified in failure_index. If the failure cannot be attributed to a given attribute, failure_index contains -1. If an attribute instance already exists which is identical in both attr_id and attr_value to an attribute specified in in_attrs, the existing attribute information is overwritten by the new information. For multi-valued attributes, every instance with the same attr_id is overwritten with the supplied values. If an attribute instance does not exist, it is created. If you specify ...
libc.a referencelibc.a reference
unsigned int attr; if ( !_dos_getfileattr("FOO.DAT", &attr) ) { puts("FOO.DAT attributes are:"); if ( attr & _A_ARCH ) puts("Archive"); if ( attr & _A_RDONLY ) puts("Read only"); if ( attr & _A_HIDDEN ) puts("Hidden"); if ( attr & _A_SYSTEM ) puts("Is it part of DOS ?"); if ( attr & _A_VOLID ) puts("Volume ID"); if ( attr & _A_SUBDIR ) puts("Directory"); } else puts("Unable to get FOO.DAT attributes ...
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Toxic polyneuropathy -- Vincristine Symptoms, Diagnosis, Treatments and Causes - RightDiagnosis.comToxic polyneuropathy -- Vincristine Symptoms, Diagnosis, Treatments and Causes - RightDiagnosis.com
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Polyneuropathy from small amounts of vitamins, b6 b1 b3 ...: loss of feeling in feet, pain, tingling | Page 3 | Phoenix Rising...Polyneuropathy from small amounts of vitamins, b6 b1 b3 ...: loss of feeling in feet, pain, tingling | Page 3 | Phoenix Rising...
@Gondwanaland Same question to you: how does your polyneuropathy do since? same? improved? Any treartment ideas? is yours axonal or myelin-related?...
The 1997 British Informatics OlympiadThe 1997 British Informatics Olympiad
You are to write a program that will fit a set of dominoes onto a grid which is 7 squares high, and eight squares across. This is to be subject to restrictions, in the form of doubles in fixed positions, and to the rule that two touching squares can only be equal if they belong to the same domino. A square should only be considered to touch the four squares directly above, below, left or right. Dominoes may not overlap. You should first read in input indicating where the fixed doubles are. For each double this will be in the form of an x co-ordinate and y co-ordinate for one of the halves of the domino, and a R or B to indicate whether the other half is to the right or below. The top left corner of the board is at (1,1). The list of doubles will terminate with the number -1. Note that not all the doubles may be fixed, and that their value is left for you to decide. Once you have read the list you should try and fit the other dominoes to the grid. You should then output a valid solution if there ...
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Checkbox and select multiple documents in Web Domino View (with pressing button)Checkbox and select multiple documents in Web Domino View (with pressing button)
I have a domino view ProjectTasks (web) which is embedded on the main Project form. It has an Add task button and Delete task button. In the first column of my view, I have the following formula: I...
Developers Applaud Lotus Road MapDevelopers Applaud Lotus Road Map
The Domino developer community is pleased with the plan to support Domino while developing next-generation Java technologies, but concerns remain.
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Your bodys many systems work closely together to maintain optimal health, so when one system is off balance it can trigger a domino effect, potentially...
Weiterbildungs-DetailWeiterbildungs-Detail
$(document).ready(function() { $(.tabLink).each(function(){ $(this).click(function(){ tabeId = $(this).attr(id); $(.tabLink).removeClass(active...
Hereditary gelsolin amyloidosisHereditary gelsolin amyloidosis
List of MeSH codes (C10)List of MeSH codes (C10)
List of MeSH codes (C18)List of MeSH codes (C18)
List of MeSH codes (C16)List of MeSH codes (C16)
Neuropathy (disambiguation)Neuropathy (disambiguation)
Familial amyloid neuropathyFamilial amyloid neuropathy
Carpal tunnel syndromeCarpal tunnel syndrome
Lattice corneal dystrophyLattice corneal dystrophy
Familial amyloid cardiomyopathyFamilial amyloid cardiomyopathy
Multiple myelomaMultiple myeloma
Bells palsyBell's palsy
Complex regional pain syndromeComplex regional pain syndrome
Meralgia paraestheticaMeralgia paraesthetica
AL amyloidosisAL amyloidosis
Dejerine-Sottas diseaseDejerine-Sottas disease
TafamidisTafamidis
Demyelinating diseaseDemyelinating disease
Guillain-Barré syndromeGuillain-Barré syndrome
Tarsal tunnel syndromeTarsal tunnel syndrome
Ulnar nerve entrapmentUlnar nerve entrapment
ICD-10 Chapter VI: Diseases of the nervous systemICD-10 Chapter VI: Diseases of the nervous system
TransthyretinTransthyretin
Charcot-Marie-Tooth diseaseCharcot-Marie-Tooth disease
List of OMIM disorder codesList of OMIM disorder codes
AmyloidosisAmyloidosis
Transthyretin-related hereditary amyloidosisTransthyretin-related hereditary amyloidosis
Ardalan-Shoja-Kiuru syndromeArdalan-Shoja-Kiuru syndrome
List of diseases (C)List of diseases (C)
AmylinAmylin
List of MeSH codes (C14)List of MeSH codes (C14)
Epigenetics of neurodegenerative diseasesEpigenetics of neurodegenerative diseases
RNA interferenceRNA interference
SOD1SOD1
Cardiac amyloidosisCardiac amyloidosis
User:JfdwolffUser:Jfdwolff
SOD1SOD1
ICD-10 Chapter IV: Endocrine, nutritional and metabolic diseasesICD-10 Chapter IV: Endocrine, nutritional and metabolic diseases
List of cutaneous conditionsList of cutaneous conditions
AnatomyOrganismsDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPsychiatry and PsychologyPhenomena and ProcessesDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaHumanitiesInformation ScienceNamed GroupsHealth CareGeographicals
Amyloid NeuropathiesAmyloid Neuropathies, FamilialPrealbuminAmyloidosisAmyloidDiabetic NeuropathiesAmyloid beta-PeptidesSerum Amyloid A ProteinPeripheral Nervous System DiseasesAmyloid beta-Protein PrecursorPlaque, AmyloidHereditary Sensory and Motor NeuropathyIslet Amyloid PolypeptideCerebral Amyloid AngiopathyHereditary Sensory and Autonomic NeuropathiesOptic Neuropathy, IschemicSerum Amyloid P-ComponentPolyneuropathiesSural NerveAmyloid Precursor Protein SecretasesAlzheimer DiseaseDiflunisalLiver TransplantationComplicityEroticaOligonucleotidesRNAMolecular Sequence DataBase SequenceOligonucleotides, AntisenseAmyloidosis, FamilialHistology, ComparativeCryptococcus gattiiMass SpectrometrySwedenFaculty, MedicalFacultyTandem Mass SpectrometryParaphiliasFaculty, DentalComplement ActivationComplement Pathway, ClassicalComplement C3Syncope, VasovagalTilt-Table TestSyncopeOrthostatic IntoleranceAtrial Natriuretic FactorChemistry, AnalyticHypotension, OrthostaticFlatulenceInstitutionalizationHeartburnDrug-Related Side Effects and Adverse ReactionsDiagnostic Techniques and ProceduresAbbreviations as TopicDigestive System
Familial amyloid neuropathy - WikipediaFamilial amyloid neuropathy - Wikipedia
Marktanalyse - Familial Amyloid Neuropathies - Pipeline Review, H2 2016Marktanalyse - Familial Amyloid Neuropathies - Pipeline Review, H2 2016
Familial Amyloid Neuropathies - Pipeline Review| Therapeutics Development by Leading Key Players: Alnylam Pharmaceuticals,...Familial Amyloid Neuropathies - Pipeline Review| Therapeutics Development by Leading Key Players: Alnylam Pharmaceuticals,...
Tongue atrophy and fasciculations in transthyretin familial amyloid neuropathy | Neurology GeneticsTongue atrophy and fasciculations in transthyretin familial amyloid neuropathy | Neurology Genetics
Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.govEfficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy - Full Text View - ClinicalTrials.gov
Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis Corino de Andrades Disease) Ongoing Global Clinical Trials Analysis...Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis Corino de Andrade's Disease) Ongoing Global Clinical Trials Analysis...
Familial Amyloid Polyneuropathy Market: By Type (FAP-I, FAP-II, FAP-III, and FAP-IV), By Category (Peripheral Sensorimotor...Familial Amyloid Polyneuropathy Market: By Type (FAP-I, FAP-II, FAP-III, and FAP-IV), By Category (Peripheral Sensorimotor...
Compound Report CardCompound Report Card
Transthyretin amyloidosis: MedlinePlus GeneticsTransthyretin amyloidosis: MedlinePlus Genetics
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated...APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated...
Disphosphonates cardiac uptake in familial amyloid neuropathy: Comparison between DPD and HMDP | Orphanet Journal of Rare...Disphosphonates cardiac uptake in familial amyloid neuropathy: Comparison between DPD and HMDP | Orphanet Journal of Rare...
Familial amyloidosis definition | Drugs.comFamilial amyloidosis definition | Drugs.com
HDL Acute Lipid Optimization in Homozygous Familial HypercholesterolemiaHDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia
CLINICAL EVALUATION AND INVESTIGATION OF NEUROPATHY | Journal of Neurology, Neurosurgery & PsychiatryCLINICAL EVALUATION AND INVESTIGATION OF NEUROPATHY | Journal of Neurology, Neurosurgery & Psychiatry
Cardiac amyloidosis: An update on pathophysiology, diagnosis, and treatmentCardiac amyloidosis: An update on pathophysiology, diagnosis, and treatment
Bachem - Oligonucleotide Trends January 2020Bachem - Oligonucleotide Trends January 2020
Transthyretin-Related Amyloidosis Follow-up: Complications, PrognosisTransthyretin-Related Amyloidosis Follow-up: Complications, Prognosis
TNFRSF10C Gene - GeneCards | TR10C Protein | TR10C AntibodyTNFRSF10C Gene - GeneCards | TR10C Protein | TR10C Antibody
Global Bioresorbable Scaffolds IndustryGlobal Bioresorbable Scaffolds Industry
Asia Pacific Brain Aneurysm Treatment Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Type, Condition, End...Asia Pacific Brain Aneurysm Treatment Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Type, Condition, End...
Pfizers Vyndaqel® (tafamidis) First Therapy Approved in the European Union for the Rare and Fatal Neurodegenerative Disease...Pfizer's Vyndaqel® (tafamidis) First Therapy Approved in the European Union for the Rare and Fatal Neurodegenerative Disease...
United States Transthyretin Market Report 2017 : ReportsnReportsUnited States Transthyretin Market Report 2017 : ReportsnReports
Amyloid NeuropathyAmyloid Neuropathy
Wheeless Textbook of OrthopaedicsWheeless' Textbook of Orthopaedics
Open-Label Study Evaluating Synera® in the Treatment of Patients With Carpal Tunnel SyndromeOpen-Label Study Evaluating Synera® in the Treatment of Patients With Carpal Tunnel Syndrome
Immunoaffinity chromatographic and immunoprecipitation methods combined with mass spectrometry for characterization of...Immunoaffinity chromatographic and immunoprecipitation methods combined with mass spectrometry for characterization of...
Late onset lattice corneal dystrophy with systemic familial amyloidosis, amyloidosis V, in an English family | British Journal...Late onset lattice corneal dystrophy with systemic familial amyloidosis, amyloidosis V, in an English family | British Journal...
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What Price Success? Ionis Drug Worked in Phase 3 but Had Serious Side Effects | ALZFORUMWhat Price Success? Ionis Drug Worked in Phase 3 but Had Serious Side Effects | ALZFORUM
The differential diagnosis of neurogenic and referred leg painThe differential diagnosis of neurogenic and referred leg pain
CiNii Articles - Tojo KanaCiNii Articles - Tojo Kana
Anti-Gelsolin antibody - C-terminal (ab191134) | AbcamAnti-Gelsolin antibody - C-terminal (ab191134) | Abcam
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Vasoactive Intestinal Peptide (Vip) AntikörperVasoactive Intestinal Peptide (Vip) Antikörper
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Computational and Mathematical Methods in MedicineComputational and Mathematical Methods in Medicine
Read eBooks online | World Heritage Encyclopedia | Apolipoprotein A1Read eBooks online | World Heritage Encyclopedia | Apolipoprotein A1
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Rare Diseases Awareness Ribbons - A | Personalized CauseRare Diseases Awareness Ribbons - A | Personalized Cause
Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 : ReportsnReportsTransthyretin (ATTR or Prealbumin or TBPA or TTR) - Pipeline Review, H2 2017 : ReportsnReports
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Autonomic description in patients with very early TTR amyloidosis (Familial Amyloid Polyneuropathy). (P2.115) | NeurologyAutonomic description in patients with very early TTR amyloidosis (Familial Amyloid Polyneuropathy). (P2.115) | Neurology
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Hereditary Transthyretin amyloidosis | Amyloidosis AllianceHereditary Transthyretin amyloidosis | Amyloidosis Alliance
s ꒲ | [ g: g X T C ` F p C v C i ̕s ꒲ | [ g: g X T C ` F p C v C i ̕
Is Your Disease on the RARE List™ - If So, More Bad News! | The Addi and Cassi Fund - Niemann Pick Type CIs Your Disease on the RARE List™ - If So, More Bad News! | The Addi and Cassi Fund - Niemann Pick Type C
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Demyelinating disease - WikipediaDemyelinating disease - Wikipedia
Bells palsy - WikipediaBell's palsy - Wikipedia
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藥學系 - 研究成果 - 臺北醫學大學藥學系 - 研究成果 - 臺北醫學大學
Emed.co.il | ???? ??? ?????? ??? - ?????? ???? ??? ??? AMYLOIDOSIS CONFERENCE ?????? - 28.3.2019Emed.co.il | ???? ??? ?????? ??? - ?????? ???? ??? ??? AMYLOIDOSIS CONFERENCE ?????? - 28.3.2019
Emed.co.il | ???? ??? ?????? ??? - ?????? ???? ??? ??? AMYLOIDOSIS CONFERENCE ?????? - 28.3.2019Emed.co.il | ???? ??? ?????? ??? - ?????? ???? ??? ??? AMYLOIDOSIS CONFERENCE ?????? - 28.3.2019
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Search Articles | University of Toronto LibrariesSearch Articles | University of Toronto Libraries
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Search | Global Index MedicusSearch | Global Index Medicus
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Volume 3, No 4 - Fall 2006 | MedReviewsVolume 3, No 4 - Fall 2006 | MedReviews
Neuropathies | definition of neuropathies by Medical dictionaryNeuropathies | definition of neuropathies by Medical dictionary
Autonomic Peripheral Neuropathy | Wackbag.comAutonomic Peripheral Neuropathy | Wackbag.com
Autoimmune peripheral neuropathy | definition of Autoimmune peripheral neuropathy by Medical dictionaryAutoimmune peripheral neuropathy | definition of Autoimmune peripheral neuropathy by Medical dictionary
AnatomyOrganismsDiseasesChemicals and DrugsAnalytical, Diagnostic and Therapeutic Techniques and EquipmentPsychiatry and PsychologyPhenomena and ProcessesDisciplines and OccupationsAnthropology, Education, Sociology and Social PhenomenaHumanitiesInformation ScienceNamed GroupsHealth CareGeographicals
Amyloid NeuropathiesAmyloid Neuropathies, FamilialPrealbuminAmyloidosisAmyloidDiabetic NeuropathiesAmyloid beta-PeptidesSerum Amyloid A ProteinPeripheral Nervous System DiseasesAmyloid beta-Protein PrecursorPlaque, AmyloidHereditary Sensory and Motor NeuropathyIslet Amyloid PolypeptideCerebral Amyloid AngiopathyHereditary Sensory and Autonomic NeuropathiesOptic Neuropathy, IschemicSerum Amyloid P-ComponentPolyneuropathiesSural NerveAmyloid Precursor Protein SecretasesAlzheimer DiseaseDiflunisalLiver TransplantationComplicityEroticaOligonucleotidesRNAMolecular Sequence DataBase SequenceOligonucleotides, AntisenseAmyloidosis, FamilialHistology, ComparativeCryptococcus gattiiMass SpectrometrySwedenFaculty, MedicalFacultyTandem Mass SpectrometryParaphiliasFaculty, DentalComplement ActivationComplement Pathway, ClassicalComplement C3Syncope, VasovagalTilt-Table TestSyncopeOrthostatic IntoleranceAtrial Natriuretic FactorChemistry, AnalyticHypotension, OrthostaticFlatulenceInstitutionalizationHeartburnDrug-Related Side Effects and Adverse ReactionsDiagnostic Techniques and ProceduresAbbreviations as TopicDigestive System
AmyloidosisFibrilsAutonomic neuropathyDepositsSensoryCardiacCardiomyopathyDiagnosisPolyneuropathiesTreatment of familial amyloid polyneCorneal dystrophyDiabetic NeuropathyGeneticSystemicAutosomalGelsolinMutationsDisordersPrevalenceSyndromeProteinsSymptomsDiseaseAmyloidotic neuropathiesClinicalProtein called amyloidVasculiticFibril proteinInvolvementMutationPrecursorRenalPatientsSmall-fiber neuropathyNeuropathicProgressionLiverTissueEntrapmentDysfunction
Amyloidosis45
Fibrils14
Autonomic neuropathy11
Deposits21
Sensory19
Cardiac4
Cardiomyopathy3
Diagnosis6
Polyneuropathies1
Treatment of familial amyloid polyne2
Corneal dystrophy1
Diabetic Neuropathy4
Genetic6
Systemic4
Autosomal3
Gelsolin1
Mutations2
Disorders14
Prevalence4
Syndrome4
Proteins2
Symptoms2
Disease16
Amyloidotic neuropathies2
Clinical2
  • The assessment and investigation of a possible neuropathy is one of the most common clinical problems facing the general neurologist. (bmj.com)
  • We examined the DNA analysis of familial amyloid polyneuropathy (FAP) patients and their families from Nagano and Hiroshima prefectures in Japan using recombinant DNA techniques and compared the results with the clinical features. (elsevier.com)
Protein called amyloid1
Vasculitic2
Fibril protein2
Involvement2
  • Involvement of cranial nerves (for example, facial numbness or weakness, oculomotor disturbance) in an acute inflammatory neuropathy is helpful in excluding a cord lesion with a pseudo-lower motor neurone pattern of presentation, as may occur in acute myelopathies. (bmj.com)
  • Few peripheral neuropathies are associated with pure or predominantly small fiber involvement. (medscape.com)
Mutation1
Precursor3
Renal2
Patients15
Small-fiber neuropathy1
  • A parallel focus in this laboratory is to use the previously described technique to diagnose human neuropathies involving small-diameter nociceptive nerves (small-fiber neuropathy). (ntu.edu.tw)
Neuropathic1
Progression1
Liver1
Tissue1
Entrapment1
Dysfunction2
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated...
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Transthyretin amyloidosis: MedlinePlus Genetics
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TNFRSF10C Gene - GeneCards | TR10C Protein | TR10C Antibody
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Familial Amyloid Polyneuropathy (Transthyretin Amyloidosis Corino de Andrade's Disease) Ongoing Global Clinical Trials Analysis...
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Global Bioresorbable Scaffolds Industry
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Familial Amyloid Polyneuropathy Market: By Type (FAP-I, FAP-II, FAP-III, and FAP-IV), By Category (Peripheral Sensorimotor...
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CLINICAL EVALUATION AND INVESTIGATION OF NEUROPATHY | Journal of Neurology, Neurosurgery & Psychiatry
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Table of Contents - February 01, 1988, 38 (2) | Neurology
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Abnormal small bowel motility in patients with hereditary transthyretin amyloidosis
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Complement activation in acquired and hereditary amyloid neuropathy<...
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Amyloidosis, Finnish Type disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
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Dr. Sung-Tsang Hsieh
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Autonomic Nervous System Anatomy: Overview, Gross Anatomy, Cardiac and Vascular Regulation
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Digestive symptoms - RightDiagnosis.com
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Neuropeptides and peptide hormones in syncope and orthostatic intolerance | Krishnan | Cardiology Journal
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Andras Khoor, MD - Research Output - Mayo Clinic
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Anti-Gelsolin antibody - C-terminal (ab191134) | Abcam
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Recombinant Anti-Gelsolin antibody [EPR1942] - BSA and Azide free (ab236029)
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Familial Amyloid Polyneuropathy Market Is Estimated to Expand at a Robust CAGR over 2020 | Jun 22, 2018 - ReleaseWire
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Amyloidosis Types | Amyloidosis Center
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Autonomic Failure: A Textbook of Clinical Disorders of the Autonomic Nervous System - Oxford Medicine
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Is Your Disease on the RARE List™ - If So, More Bad News! | The Addi and Cassi Fund - Niemann Pick Type C
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Is CRISPR a Solution to Familial Amyloid Polyneuropathy? | Leaders in Pharmaceutical Business Intelligence (LPBI) Group
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Info On Gout | Psoriatic Arthritis Symptoms
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Mold Gout | In Search of Healers for Joint Pain
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Frequent genes in rare diseases: panel-based next generation sequencing to disclose causal mutations in hereditary neuropathies...
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What is amyloid arthropathy?
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Disphosphonates cardiac uptake in familial amyloid neuropathy: Comparison between DPD and HMDP | Orphanet Journal of Rare...
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