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*  Creon (Pancrelipase Capsules): Side Effects, Interactions, Warning, Dosage & Uses
Patients received their usual pancreatic enzyme replacement therapy (mean dose of 7,000 lipase units/kg/day for a mean duration ... Patients received their usual pancreatic enzyme replacement therapy (mean dose of 7,000 lipase units/kg/day for a mean duration ... When patients were switched from their usual pancreatic enzyme replacement therapy to CREON, they demonstrated similar spot ... Dosage recommendations for pancreatic enzyme replacement therapy were published following the Cystic Fibrosis Foundation ...
  https://www.rxlist.com/creon-drug.htm
*  Hunter syndrome - Wikipedia
Since the implementation of enzyme replacement therapy for Hunter syndrome, lifespans for those without mental handicaps are ... a clinical review and recommendations for treatment in the era of enzyme replacement therapy". Eur. J. Pediatr. 167 (3): 267-77 ... possibly even equivalent to weekly enzyme replacement therapy, resulting in much improved life expectancy. However, even for ... "A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)". ...
  https://en.wikipedia.org/wiki/Hunter_syndrome
*  Mucopolysaccharidosis type II - Genetics Home Reference
... a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008 Mar;167(3): ... Mutations in the IDS gene reduce or completely eliminate the function of the I2S enzyme. Lack of I2S enzyme activity leads to ... The IDS gene provides instructions for producing the I2S enzyme, which is involved in the breakdown of large sugar molecules ...
  https://ghr.nlm.nih.gov/condition/mucopolysaccharidosis-type-ii
*  Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support - Full...
patient has received enzyme replacement therapy with GAA from any source. *patient has taken an experimental drug in the 30 ... Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body ...
  https://clinicaltrials.gov/ct2/show/NCT00268944?term=Pompe&rank=13
*  A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe...
Patient has received enzyme replacement therapy with GAA from any source;. *Patient has used an investigational product within ... Cardiovascular abnormalities in late-onset Pompe disease and response to enzyme replacement therapy. Genet Med. 2011 Jul;13(7): ... Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body ...
  https://clinicaltrials.gov/show/NCT00158600
*  Hunter Syndrome (Mucopolysaccharidosis Type II): Background, Pathophysiology, Epidemiology
The MPSs are caused by a deficiency of lysosomal enzymes required for the degradation of mucopolysaccharides or ... Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II. J Inherit Metab Dis. 2011 Feb. ... Mucopolysaccharidosis type II in females and response to enzyme replacement therapy. Am J Med Genet A. 2012 Feb. 158A(2):450-4 ... Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy ...
  https://emedicine.medscape.com/article/944723-overview
*  Systemic disease and the heart: Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective...
Evidence showing that enzyme-replacement therapy (ERT) can be effective in reducing LV hypertrophy and improving myocardial ... Home » Systemic disease and the heart: Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a ... Systemic disease and the heart: Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective ... Conclusions: Long-term therapy with agalsidase beta at 1 mg/kg every 2 weeks was effective in significantly reducing LV ...
  http://connection.ebscohost.com/c/articles/43671861/systemic-disease-heart-effects-enzyme-replacement-therapy-patients-anderson-fabry-disease-prospective-long-term-cardiac-magnetic-resonance-imaging-study
*  Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease - Full...
... as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with Classical Infantile Pompe disease who have ... Pompe disease is caused by a deficiency of a critical enzyme in the body called acid alpha glucosidase (GAA). Normally, GAA is ... a small, but inactive, amount of natural GAA enzyme present in their bodies (called Cross-Reacting Immunologic Material- ...
  https://clinicaltrials.gov/ct2/show/NCT00025896?cond=%22glycogen+storage+disease+type+IX%22+OR+%22Glycogen+Storage+Disease%22&rank=14
*  Mucopolysaccharidosis I (MPS I) Registry - Full Text View - ClinicalTrials.gov
... the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy ... Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme ...
  https://clinicaltrials.gov/ct2/show/NCT00144794
*  Diagnosing Lysosomal Storage Disorders: Mucopolysaccharidosis Type II - Current Protocols
A clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur. J. Pediatr. 167:267‐277. ... Twelve different enzyme assays on dried‐blood filter paper samples for detection of patients with selected inherited lysosomal ... A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease). J. Inherit. Metab. Dis. 24:675‐680. ... The advent of fluorometric and mass spectrometry methods for enzyme analysis in DBS has simplified the diagnostic approach for ...
  http://www.currentprotocols.com/WileyCDA/CPUnit/refId-hg1714.html?quicktabs_cp=materials
*  A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II - Full Text View -...
... as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must ... as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II).. ... GSD-II (also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase ( ...
  https://clinicaltrials.gov/ct2/show/NCT00051935?cond=%22Glycogen+storage+disease+type+2%22&rank=2
*  Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus...
This model can potentially be expanded to include the treatment of other lysosomal enzyme disorders. Lessons learned from ... systemic genetic therapy of muscle disorders also should have implications for other muscle diseases, such as the muscular ... This report demonstrates that a single intravenous administration of a gene therapy vector can potentially result in the ... on the positive attributes of modified adenovirus-based vectoring systems and receptor-mediated lysosomal targeting of enzymes ...
  https://www.semanticscholar.org/paper/Systemic-correction-of-the-muscle-disorder-glycoge-Amalfitano-McVie-Wylie/bf9b8d849d8f2d9c3a2dbd22fe3ef88b80b6ac32
*  A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease - Full Text View - ClinicalTrials.gov
... as a potential enzyme replacement therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are less ... Pompe disease (also known as glycogen storage disease type II, 'GSD-II') is caused by a deficiency of a critical enzyme in the ...
  https://clinicaltrials.gov/ct2/show/NCT00059280?term=Pompe+Disease&rank=18
*  Glycogen storage disease type II - Wikipedia
In a study which included the largest cohort of patients with Pompe disease treated with enzyme replacement therapy (ERT) to ... This was based on enzyme replacement therapy using biologically active recombinant human alglucosidase alfa produced in Chinese ... He joined the company Novazyme in 1999, which was working on enzyme replacement treatment for Pompe's. Novazyme was sold to ... Myozyme is FDA approved for replacement therapy for infantile-onset Pompe disease. Recent studies on chaperone molecules to be ...
  https://en.wikipedia.org/wiki/Glycogen_storage_disease_type_II
*  FDA clears Alexion's enzyme replacement therapy Kanuma - PharmaTimes
US regulators have issued a green light for Alexion's long-term enzyme replacement therapy Kanuma, giving patients with the ... US regulators have issued a green light for Alexion's long-term enzyme replacement therapy Kanuma, giving patients with the ... Alexion's enzyme replacement therapy for another ultra-rare condition called paediatric-onset hypophosphatasia. ...
  http://www.pharmatimes.com/news/fda_clears_alexions_enzyme_replacement_therapy_kanuma_972250
*  Enzyme Replacement Therapy in Fabry Disease - Full Text View - ClinicalTrials.gov
This study wants to evaluate the long term efficacy of enzyme replacement therapy in patients with Fabry disease and renal ... Evaluation of the Long Term Efficacy of Enzyme Replacement Therapy in Fabry Disease. ... Infusions of the enzyme replacement treatment have been demonstrated to be safe and effective. ... In the classic form (where the enzyme activity is absent) the clinical findings are represented by pain and paresthesias in the ...
  https://clinicaltrials.gov/show/NCT00149318
*  Muscle Response to Enzyme Replacement Therapy in Pompe Disease - Tabular View - ClinicalTrials.gov
Muscle Response to Enzyme Replacement Therapy in Pompe Disease. Official Title Muscle Response to Enzyme Replacement Therapy in ... Muscle Response to Enzyme Replacement Therapy in Pompe Disease. This study is currently recruiting participants. See Contacts ... This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy. ...
  https://clinicaltrials.gov/ct2/show/record/NCT00713453
*  Enzyme replacement therapy
Tifft, C., Proud, V., Levy, P., DeMarco, K., Nicely, H. and Turbeville, S. (2009) Enzyme Replacement Therapy in the Home ... Galsulfase, a Food and Drug Administration-approved enzyme replacement therapy for mucopolysaccharidosis VI, is administered ... some physicians are transitioning appropriate patients to home infusion therapy. An online survey was conducted with 3 ... 4 patients with mucopolysaccharidosis VI to better understand the factors motivating the transition to home infusion therapy, ...
  http://www.ivteam.com/intravenous-literature/enzyme-replacement-therapy/
*  Enzyme Replacement Therapy for Lysosomal Storage Disorders | Effective Health Care Program
ERT enzyme replacement therapy. GSD II glycogen storage disease type II (Pompe disease). IMD inherited metabolic disorders. LSD ... What FDA-approved enzyme replacement therapy (ERT) products are available for lysosomal storage diseases (LSD)?. *What are the ... Enzyme reconstitution/replacement therapy for lysosomal storage diseases. Curr Opin Pediatr 2007 Dec;19(6):628-35. PMID: ... Diseases with FDA-approved Enzyme Replacement Therapy*. Disease [estimated incidence]. Symptoms. FDA-Approved Product (approval ...
  https://effectivehealthcare.ahrq.gov/topics/lysosomal-storage-diseases-therapies/research-protocol
*  Enzyme replacement therapy on hypophosphatasia mouse model | Springer for Research & Development
Enzyme replacement therapy (ERT) for HPP by functional TNSALP is one of the therapeutic options. The C-terminal-anchorless ... Millán JL, Narisawa S, Lemire I et al (2008) Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res 23:777-87 ... Bone Mineral Density Enzyme Replacement Therapy Fabry Disease Homozygous Mouse Hypophosphatasia These keywords were added by ... McKee MD, Nakano Y, Masica DL et al (2011) Enzyme replacement therapy prevents dental defects in a model of hypophosphatasia. J ...
  https://rd.springer.com/article/10.1007/s10545-013-9646-7
*  Enzyme replacement therapy - Wikipedia
Protein replacement therapy Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016 ... Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. ... Neufeld EF (2006). "Chapter 10: Enzyme replacement therapy - a brief history". In Mehta A, Beck M, Sunder-Plassmann G. Fabry ... "A 24-Year Enzyme Replacement Therapy in an Adenosine-deaminase-Deficient Patient". Pediatrics. 137 (1): e20152169. doi:10.1542/ ...
  https://en.wikipedia.org/wiki/Enzyme_replacement_therapy
*  Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) - Study Results - ClinicalTrials.gov
Biological: Iduronate-2-sulfatase enzyme replacement therapy. Biological: iduronate-2-sulfatase enzyme replacement therapy. ... Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II). The safety and scientific validity of ...
  https://clinicaltrials.gov/ct2/show/results/NCT00069641
*  Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease | Circulation
Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA. 2001; 285: 2743-2749. ... Recently, a new therapeutic approach of enzyme replacement therapy (ERT) with recombinant α-Gal A was introduced.2-3 First ... Background- Enzyme replacement therapy (ERT) has been shown to enhance microvascular endothelial globotriaosylceramide ... Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease. A Prospective Strain Rate ...
  http://circ.ahajournals.org/content/108/11/1299
*  FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome - BioTech Weblog
FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome 0 By Ruth on July 26, 2006. · Drugs, Vaccines and ... You are at:Home»Drugs, Vaccines and Therapeutics»FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome ... It is given as weekly infusions, a therapy that is expected to be one of the world's most expensive medical treatments, at ... Individuals afflicted with this disorder lack an enzyme called iduronate-2-sulfatase (I2S), and are unable to break down and ...
  http://www.biotech-weblog.com/fda-approves-elaprase-enzyme-replacement-therapy-for-hunters-syndrome/
*  Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency - Patent # 8709408 - PatentGenius
The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of ... Beutler et al., 1977, "Enzyme replacement therapy in Gaucher's disease: preliminary clinical trial of a new enzyme preparation ... Pentchev, 1977, "Enzyme replacement therapy in Gaucher's and Fabry's disease", Ann. Clin. Lab. Sci. 7:251-253. cited by ... McGovern et al, 2009, "A Phase 1 Trial of Recombinant Human Acid Sphingomyelinase (rhASM) Enzyme Replacement Therapy in Adults ...
  http://www.patentgenius.com/patent/8709408.html