Muscular Diseases: Acquired, familial, and congenital disorders of SKELETAL MUSCLE and SMOOTH MUSCLE.Muscular Dystrophy, Facioscapulohumeral: An autosomal dominant degenerative muscle disease characterized by slowly progressive weakness of the muscles of the face, upper-arm, and shoulder girdle. The onset of symptoms usually occurs in the first or second decade of life. Affected individuals usually present with impairment of upper extremity elevation. This tends to be followed by facial weakness, primarily involving the orbicularis oris and orbicularis oculi muscles. (Neuromuscul Disord 1997;7(1):55-62; Adams et al., Principles of Neurology, 6th ed, p1420)Muscular Dystrophies: A heterogeneous group of inherited MYOPATHIES, characterized by wasting and weakness of the SKELETAL MUSCLE. They are categorized by the sites of MUSCLE WEAKNESS; AGE OF ONSET; and INHERITANCE PATTERNS.Muscular Dystrophy, Duchenne: An X-linked recessive muscle disease caused by an inability to synthesize DYSTROPHIN, which is involved with maintaining the integrity of the sarcolemma. Muscle fibers undergo a process that features degeneration and regeneration. Clinical manifestations include proximal weakness in the first few years of life, pseudohypertrophy, cardiomyopathy (see MYOCARDIAL DISEASES), and an increased incidence of impaired mentation. Becker muscular dystrophy is a closely related condition featuring a later onset of disease (usually adolescence) and a slowly progressive course. (Adams et al., Principles of Neurology, 6th ed, p1415)Chromosomes, Human, Pair 4: A specific pair of GROUP B CHROMOSOMES of the human chromosome classification.Muscular Dystrophy, AnimalDystrophin: A muscle protein localized in surface membranes which is the product of the Duchenne/Becker muscular dystrophy gene. Individuals with Duchenne muscular dystrophy usually lack dystrophin completely while those with Becker muscular dystrophy have dystrophin of an altered size. It shares features with other cytoskeletal proteins such as SPECTRIN and alpha-actinin but the precise function of dystrophin is not clear. One possible role might be to preserve the integrity and alignment of the plasma membrane to the myofibrils during muscle contraction and relaxation. MW 400 kDa.Myotonic Dystrophy: Neuromuscular disorder characterized by PROGRESSIVE MUSCULAR ATROPHY; MYOTONIA, and various multisystem atrophies. Mild INTELLECTUAL DISABILITY may also occur. Abnormal TRINUCLEOTIDE REPEAT EXPANSION in the 3' UNTRANSLATED REGIONS of DMPK PROTEIN gene is associated with Myotonic Dystrophy 1. DNA REPEAT EXPANSION of zinc finger protein-9 gene intron is associated with Myotonic Dystrophy 2.Muscular Dystrophies, Limb-Girdle: A heterogenous group of inherited muscular dystrophy that can be autosomal dominant or autosomal recessive. There are many forms (called LGMDs) involving genes encoding muscle membrane proteins such as the sarcoglycan (SARCOGLYCANS) complex that interacts with DYSTROPHIN. The disease is characterized by progressing wasting and weakness of the proximal muscles of arms and legs around the HIPS and SHOULDERS (the pelvic and shoulder girdles).Sarcoglycans: A family of transmembrane dystrophin-associated proteins that play a role in the membrane association of the DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX.Calpain: Cysteine proteinase found in many tissues. Hydrolyzes a variety of endogenous proteins including NEUROPEPTIDES; CYTOSKELETAL PROTEINS; proteins from SMOOTH MUSCLE; CARDIAC MUSCLE; liver; platelets; and erythrocytes. Two subclasses having high and low calcium sensitivity are known. Removes Z-discs and M-lines from myofibrils. Activates phosphorylase kinase and cyclic nucleotide-independent protein kinase. This enzyme was formerly listed as EC 3.4.22.4.Dystroglycans: Dystrophin-associated proteins that play role in the formation of a transmembrane link between laminin-2 and DYSTROPHIN. Both the alpha and the beta subtypes of dystroglycan originate via POST-TRANSLATIONAL PROTEIN PROCESSING of a single precursor protein.National Academy of Sciences (U.S.): A United States organization of distinguished scientists and engineers established for the purpose of investigating and reporting upon any subject of art or science as requested by any department of government. The National Research Council organized by NAS serves as the principal operating agency to stimulate and support research.Myopathies, Nemaline: A group of inherited congenital myopathic conditions characterized clinically by weakness, hypotonia, and prominent hypoplasia of proximal muscles including the face. Muscle biopsy reveals large numbers of rod-shaped structures beneath the muscle fiber plasma membrane. This disorder is genetically heterogeneous and may occasionally present in adults. (Adams et al., Principles of Neurology, 6th ed, p1453)TexasAppointments and Schedules: The different methods of scheduling patient visits, appointment systems, individual or group appointments, waiting times, waiting lists for hospitals, walk-in clinics, etc.Neuromuscular Diseases: A general term encompassing lower MOTOR NEURON DISEASE; PERIPHERAL NERVOUS SYSTEM DISEASES; and certain MUSCULAR DISEASES. Manifestations include MUSCLE WEAKNESS; FASCICULATION; muscle ATROPHY; SPASM; MYOKYMIA; MUSCLE HYPERTONIA, myalgias, and MUSCLE HYPOTONIA.Tropomodulin: An actin capping protein that binds to the pointed-end of ACTIN. It functions in the presence of TROPOMYOSIN to inhibit microfilament elongation.National Institute of Dental and Craniofacial Research (U.S.): Component of the NATIONAL INSTITUTES OF HEALTH. It seeks to improve oral, dental and craniofacial health through research, research training, and the dissemination of health information by conducting and supporting basic and clinical research. It was established in 1948 as the National Institute of Dental Research and re-named in 1998 as the National Institute of Dental and Craniofacial Research.Nobel PrizePoliomyelitis: An acute infectious disease of humans, particularly children, caused by any of three serotypes of human poliovirus (POLIOVIRUS). Usually the infection is limited to the gastrointestinal tract and nasopharynx, and is often asymptomatic. The central nervous system, primarily the spinal cord, may be affected, leading to rapidly progressive paralysis, coarse FASCICULATION and hyporeflexia. Motor neurons are primarily affected. Encephalitis may also occur. The virus replicates in the nervous system, and may cause significant neuronal loss, most notably in the spinal cord. A rare related condition, nonpoliovirus poliomyelitis, may result from infections with nonpoliovirus enteroviruses. (From Adams et al., Principles of Neurology, 6th ed, pp764-5)Awards and PrizesHistory, 20th Century: Time period from 1901 through 2000 of the common era.History, 19th Century: Time period from 1801 through 1900 of the common era.History, 21st Century: Time period from 2001 through 2100 of the common era.Allergy and Immunology: A medical specialty concerned with the hypersensitivity of the individual to foreign substances and protection from the resultant infection or disorder.Mice, Inbred mdx: A strain of mice arising from a spontaneous MUTATION (mdx) in inbred C57BL mice. This mutation is X chromosome-linked and produces viable homozygous animals that lack the muscle protein DYSTROPHIN, have high serum levels of muscle ENZYMES, and possess histological lesions similar to human MUSCULAR DYSTROPHY. The histological features, linkage, and map position of mdx make these mice a worthy animal model of DUCHENNE MUSCULAR DYSTROPHY.Utrophin: An autosomally-encoded 376-kDa cytoskeletal protein that is similar in structure and function to DYSTROPHIN. It is a ubiquitously-expressed protein that plays a role in anchoring the CYTOSKELETON to the PLASMA MEMBRANE.Muscle, Skeletal: A subtype of striated muscle, attached by TENDONS to the SKELETON. Skeletal muscles are innervated and their movement can be consciously controlled. They are also called voluntary muscles.Earth Sciences: Fields of science encompassing studies and research from the disciplines of PHYSICS; CHEMISTRY; BIOLOGICAL SCIENCES; and MATHEMATICS; that are related to the planet EARTH. Subfields include atmospheric chemistry; CLIMATOLOGY; ENVIRONMENTAL SCIENCES; GEOGRAPHY; GEOLOGY; geophysics; METEOROLOGY; OCEANOGRAPHY; PALEONTOLOGY; mineralogy; and seismology.Science: The study of natural phenomena by observation, measurement, and experimentation.Physics: The study of those aspects of energy and matter in terms of elementary principles and laws. (From McGraw-Hill Dictionary of Scientific and Technical Terms, 6th ed)Internet: A loose confederation of computer communication networks around the world. The networks that make up the Internet are connected through several backbone networks. The Internet grew out of the US Government ARPAnet project and was designed to facilitate information exchange.Portal Vein: A short thick vein formed by union of the superior mesenteric vein and the splenic vein.Wounds and Injuries: Damage inflicted on the body as the direct or indirect result of an external force, with or without disruption of structural continuity.Nuclear Physics: The study of the characteristics, behavior, and internal structures of the atomic nucleus and its interactions with other nuclei. (From McGraw-Hill Dictionary of Scientific and Technical Terms, 6th ed)BrazilCell Nucleus: Within a eukaryotic cell, a membrane-limited body which contains chromosomes and one or more nucleoli (CELL NUCLEOLUS). The nuclear membrane consists of a double unit-type membrane which is perforated by a number of pores; the outermost membrane is continuous with the ENDOPLASMIC RETICULUM. A cell may contain more than one nucleus. (From Singleton & Sainsbury, Dictionary of Microbiology and Molecular Biology, 2d ed)PortugalMuscles: Contractile tissue that produces movement in animals.United StatesEpidemiologic Methods: Research techniques that focus on study designs and data gathering methods in human and animal populations.Morpholinos: Synthetic analogs of NUCLEIC ACIDS composed of morpholine ring derivatives (MORPHOLINES) linked by phosphorodimidates. One standard DNA nucleic acid base (ADENINE; GUANINE; CYTOSINE; OR THYMINE) is bound to each morpholine ring.Chief Executive Officers, Hospital: Individuals who have the formal authority to manage a hospital, including its programs and services, in accordance with the goals and objectives established by a governing body (GOVERNING BOARD).Hospital Administrators: Managerial personnel responsible for implementing policy and directing the activities of hospitals.MorpholinesRespiratory Muscles: These include the muscles of the DIAPHRAGM and the INTERCOSTAL MUSCLES.Genetic Therapy: Techniques and strategies which include the use of coding sequences and other conventional or radical means to transform or modify cells for the purpose of treating or reversing disease conditions.International Cooperation: The interaction of persons or groups of persons representing various nations in the pursuit of a common goal or interest.Tracheostomy: Surgical formation of an opening into the trachea through the neck, or the opening so created.Wheelchairs: Chairs mounted on wheels and designed to be propelled by the occupant.
  • With the global Orphan Drug market expected to grow to $127 billion by 2018 *, and another Rare Disease Day just having passed, there is a constantly growing awareness and interest for this industry, enabling more research to make a difference in patients' lives. (smi-online.co.uk)
  • The global spinal muscular atrophy market size was valued at US$ 884 million in 2017 and is expected to witness a robust CAGR of 12.4% over the forecast period (2018-2026). (coherentmarketinsights.com)
  • According to SMA Foundation 2015 statistics, spinal muscular atrophy has generally been believed to affect as many as 10,000 to 25,000 adults and children in the U.S. In March 2018, CureSMA stated that SMA affects around 1 in 11,000 children, and 1 in 50 is a carrier of the disease. (coherentmarketinsights.com)
  • SMi is delighted to announce the return of their 7th annual Orphan Drugs and Rare Diseases conference to London on the 18th -19th October 2017 ! (smi-online.co.uk)
  • SMi's Orphan Drugs and Rare Diseases conference will once again bring together some of the leading senior industry professionals to network, learn, and discuss current issues in the rare disease industry. (smi-online.co.uk)
  • The 7th annual Orphan Drugs and Rare Diseases conference will help connect industry professionals internationally, ensuring there is a great networking and learning opportunity to share knowledge of research, developments and issues. (smi-online.co.uk)
  • Join the Orphan Drugs and Rare Diseases community this year in October to keep up to date with the industry and to learn from our expert speaker panel, bringing you important new case studies and reports on this year's relevant topics. (smi-online.co.uk)
  • The commissioner of the Food and Drug Administration questions whether the right financial incentives are in place for drugmakers who develop orphan drugs for rare diseases. (news-medical.net)
  • The following summary is from Orphanet , a European reference portal for information on rare diseases and orphan drugs. (nih.gov)
  • Congenital myasthenic syndromes (CMS) form a heterogeneous group of rare diseases characterized by fatigable muscle weakness. (iospress.com)
  • The MHC database - The Mun-H-Center database on oral health and orofacial function in rare diseases. (mun-h-center.se)
  • We are a Swedish orofacial (mouth and face) resource center for rare diseases and are part of the specialist dental care in the Public Dental Service in the Region Västra Götaland. (mun-h-center.se)
  • Is prenatal screening for rare diseases like spinal muscular atrophy t. (bio-medicine.org)
  • Liestal, Switzerland - Santhera Pharmaceuticals (SIX:SANN), a specialty pharmaceutical company focused on the development and commercialization of innovative treatments for patients with rare diseases, announces the laun. (checkorphan.org)
  • It is one of the most common rare diseases. (coherentmarketinsights.com)
  • The eponymous label Werdnig-Hoffmann disease (sometimes misspelled with a single n) refers to the earliest clinical descriptions of childhood SMA by Johann Hoffmann and Guido Werdnig. (wikipedia.org)
  • The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases. (iospress.com)
  • CHICAGO, January 24, 2017 - The Muscular Dystrophy Association announces the award of a clinical research network grant totaling $918,000 over three years to spur advances in myotonic dystrophy (DM) research. (mda.org)
  • Ridinilazole achieved another positive Phase 2 clinical trial result, further highlighting its potential to both treat the infection and preserve the microbiome to reduce the risk of recurrent disease. (nasdaq.com)
  • Presented preclinical data at ASM Microbe 2017, including data showing that ridinilazole was very active against all 200 clinical isolates of C. difficile and was more potent than the marketed broad-spectrum antibiotics vancomycin and metronidazole. (benzinga.com)
  • These appointments, announced in May 2017, will help ensure the Company has the leadership, depth of knowledge and expertise needed to support its clinical and preclinical pipeline. (benzinga.com)
  • Vitamin D and its own analogues are trusted as remedies by clinical nephrologists particularly when treating chronic kidney disease (CKD) sufferers with extra hyperparathyroidism. (acmbcb.org)
  • He obtained his PhD in medical sciences for his neuro-oncological research in 2017 and continues to conduct clinical research in this field, currently focusing on immune therapy for glioblastoma. (ac.be)
  • Discuss the clinical manifestations and nursing management of selected disease processes and health problems. (edu.au)
  • Clinical trials using FcRn, based on work by JAX Professor Derry Roopenian , are progressing extremely well, especially for an autoimmune disease called hemolytic anemia. (jax.org)
  • Bentley alumnus Lance Colwell '92, former Vice President for the U.S. Rare Disease Group at Biogen, and Wildon Farwell, Senior Medical Director of Clinical Development at Biogen, discussed the commercial launch of the revolutionary new product, Spinraza. (bentley.edu)
  • Predicting clinical diagnosis in Huntington's disease: An imaging polymarker. (amedeo.com)
  • Some clinical trials show that carnitine supplementation is helpful for patients with angina, heart disease, or peripheral vascular disease. (mskcc.org)
  • At NeuroGen Brain and Spine Institute, stem cell treatment for muscular dystrophy has shown remarkable results in patients with the similar condition. (sooperarticles.com)
  • The mdx mice had significant tachycardia, consistent with observations in patients with muscular dystrophy. (google.com)
  • Over 50% of SMA patients die before the age of 2, and management of the disease is achieved by treating the symptoms to prevent further deterioration of the condition. (nature.com)
  • In a study more than 20 years ago, in studies supported by the Muscular Dystrophy Association, Griggs and colleagues found that a daily dose of prednisone improves patients' lives dramatically. (healthcanal.com)
  • The drug boosts muscle mass by 20 percent and slows the degeneration of the muscles in patients with the disease. (healthcanal.com)
  • Long-term disease activity and disability progression in relapsing-remitting multiple sclerosis patients on natalizumab. (nih.gov)
  • Unfortunately, an estimated 25% of these treated prostate cancer patients have been reported to progress in their disease, with cancer cells continuing to multiply within the prostate gland or having these cancer cells migrating to other parts of the body such as the lymph nodes. (belmarrahealth.com)
  • These findings resolved long-standing questions and established a disease subgroup that is ribitol-phosphate deficient, which describes a large population of dystroglycanopathy patients. (iospress.com)
  • To develop a stem cell therapy for muscular dystrophy, we are using induced pluripotent stem cells (iPSC) derived from patients, using non-integrating reprogramming methods. (stanford.edu)
  • Ezutromid is a potentially disease-modifying treatment for all patients with DMD and we look forward to reporting these initial data from this proof of concept trial. (nasdaq.com)
  • In the Kidney Disease Outcomes and Quality Initiative (KDOQI) guideline and the Kidney Disease: Improving Global Outcomes (KDIGO) guideline activated vitamin D or its analogues Gandotinib are frequently used to treat patients with secondary hyperparathyroidism and to prevent the renal osteodystrophy. (acmbcb.org)
  • Patients groups are backing gene-editing as potential "home run" against disease. (technologyreview.com)
  • Exonics will advance research underway at the University of Texas Southwestern Medical Center, where scientist Eric Olson and colleagues have cured mice of muscular dystrophy using CRISPR, stirring intense hopes among patients. (technologyreview.com)
  • Patients with muscular dystrophy lack dystrophin. (technologyreview.com)
  • The primary objective of this study is to demonstrate a pharmacodynamic effect of CK-2127107 on measures of skeletal muscle function or fatigability in patients with Spinal Muscular Atroph. (bioportfolio.com)
  • The "Sly Syndrome" name is based on William Sly, MD, who first described MPSVII and worked with a JAX team to characterize a mouse model for the disease, which affects fewer than 150 patients worldwide. (jax.org)
  • The Porteus team started with human stem cells from the blood of patients with sickle cell disease , corrected the gene mutation using CRISPR and then concentrated the human stem cells so that 90 percent carried the corrected sickle cell gene . (medicalxpress.com)
  • If the proportion of sickle cells is below 30 percent, patients have no symptoms of disease. (medicalxpress.com)
  • A stunning new breakthrough using the CRISPR-Cas9 system indicates we could potentially activate any gene to cure diseases. (inquisitr.com)
  • LA JOLLA-Salk scientists have created a new version of the CRISPR/Cas9 genome editing technology that allows them to activate genes without creating breaks in the DNA, potentially circumventing a major hurdle to using gene editing technologies to treat human diseases. (salk.edu)
  • Herein, we describe a new mouse model of this form of limb-girdle muscular dystrophy generated using CRISPR/Cas9-mediated gene editing to introduce a single thymine deletion in murine exon 6, recreating the 521ΔT point mutation in Sgcg . (biologists.org)
  • A landmark study in dogs , has succeeded in curing muscular dystrophy in four dogs by using the CRISPR Cas-9 gene-editing technique to correct mutations in the dystrophin gene. (speakingofresearch.com)
  • Though much knowledge has been acquired about ways to edit the genome using CRISPR-Cas9, there is still more work to be done before it can actually be used to cure diseases. (all-creatures.org)
  • Unfortunately, CRISPR-Cas9 is most often used to create animal models of human disease. (all-creatures.org)
  • The National Institutes of Health currently funds more than 400 protocols at an estimated cost of $100 million annually to use CRISPR-Cas9 to sicken animals with genetic defects, even though the previous NIH Director criticized scientists for "dancing around the problem" using animal models instead of directly studying human disease. (all-creatures.org)
  • A foundation representing boys dying from muscular dystrophy says it will try to cure the disease using CRISPR, a breakthrough method of correcting DNA. (technologyreview.com)
  • Because many different mutations in the dystrophin gene can lead to muscular dystrophy, initially a CRISPR treatment wouldn't fix all of them. (technologyreview.com)
  • CHICAGO, December 23, 2016 - The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza) , the first disease-modifying drug to treat the most common genetic cause of death in infants. (mda.org)
  • The per-year sticker isn't outside the range typical for rare disease treatments, which Spinraza is, but amid the intensified scrutiny on drug prices-and alongside that $750,000 tag for the first year-the announcement attracted plenty of attention. (fiercepharma.com)
  • The sticker-shock presented in the media could turn Spinraza into the Sovaldi of rare disease drugs," Porges wrote to investors after the announcement. (fiercepharma.com)
  • The research by Ivey and colleagues in 2017 compared two different participant groups who either commenced strength training or stretch control group at least 6 months post stroke . (wikibooks.org)
  • You can help advance rare disease research! (nih.gov)
  • Despite decades of research, Griggs calls the current treatment landscape for the disease "chaotic. (healthcanal.com)
  • His research interests include motor neuron disease, peripheral neuropathy and myopathy. (slu.edu)
  • As mechanisms determining the aetiology of the disease remain unclear yet, several research teams … have developed animal models from invertebrates to mammalians species. (iospress.com)
  • Shake the Disease will link state-of-the-art scientific research, visionary case studies to explore future scripts and generate new insights. (ac.be)
  • Joining principal segments of industry knowledge, precisely filed information, and solid research systems, the outcome is a research report loaded with all the information that is pivotal in fully understanding what could be possible when enhancing one's stature in the Global molecular biomarkers market research report 2017. (qyresearchreports.com)
  • The significance of the Global molecular biomarkers market research report 2017 inside the biomarkers industry is as of now a known component. (qyresearchreports.com)
  • Exclusive research philosophies are blended with demonstrated industry standard investigations to gage the regional drivers and openings inside the Global molecular biomarkers market research report 2017. (qyresearchreports.com)
  • As we definitely know the immense assorted qualities that accompany the Global molecular biomarkers market research report 2017, it is vital to recognize these distinctions with respect the purpose to capitalizing on any market circumstance. (qyresearchreports.com)
  • The competitive index in the biomarkers industry are large enough to show a seriously aggressive nature from the business sectors, and the Global molecular biomarkers market research report 2017 is no exception to this. (qyresearchreports.com)
  • We collaborated with the Muscular Dystrophy Surveillance, Tracking, and Research Network. (aappublications.org)
  • Lee, whose research has relied on the use of mouse models of disease, joined the JAX faculty in 2017. (jax.org)
  • New research from Simon John shows that adding vitamin B3 to the drinking water of mice predisposed to glaucoma can prevent the debilitating eye disease. (jax.org)
  • In the recent times, the global market for Asia pacific aspirin market report 2017 has surfaced as one of the most promising markets in the pharmaceutical industry, thanks to the significant rise in research and development activities by leading vendors of Asia pacific aspirin market report 2017 across the world. (qyresearchreports.com)
  • The research report on the Asia pacific aspirin market report 2017 is an analytical study which comprehensively analyzes the competitive framework of this market. (qyresearchreports.com)
  • The predominant applications of the Asia pacific aspirin market report 2017 have also been discussed at length in this research study. (qyresearchreports.com)
  • This kind of research, while it is still very basic, could still suggest pathways for understanding not only muscular diseases, but also some forms of cancer in which the cells also present a disorganized centrosome . (inserm.fr)
  • Discovery of the structure by scientists at Baylor College of Medicine and Rice University may help researchers learn more about a hereditary neuromuscular disease known as nemaline myopathy. (healthcanal.com)
  • LA JOLLA-Scientists have, for the first time, corrected a disease-causing mutation in early stage human embryos with gene editing. (salk.edu)
  • Salk Institute scientists have developed a novel technology to correct disease-causing aberrations in the chemical tags on DNA that affect how genes are expressed. (salk.edu)
  • In a tour de force paper published in the January 26, 2017, issue of the journal Cell, scientists at the Salk Institute report breakthroughs on multiple fronts in the race to integrate stem cells from one species into the early-stage development of another. (salk.edu)
  • It is difficult to compare the results of Dr. Mendell and colleagues with those of the phase III nusinersen study, because of the two trials' different designs, said Ans T. van der Ploeg, MD, PhD, Chair of the Center for Lysosomal and Metabolic Diseases at the Erasmus MC University in Rotterdam, the Netherlands, in an accompanying editorial. (mdedge.com)
  • More than a decade ago, the NIH's National Institute of Neurological Disorders and Stroke (NINDS) launched a special project to accelerate the translation of basic scientific discoveries into new treatments for a rare and often fatal disease. (nih.gov)
  • My area of excellence is Investigation and I am primarily focused on developing statistical methods in order to improve understanding of neurological and psychiatric diseases. (childrenshospital.org)
  • Explain the physiological processes, interactions, and controls to maintain homeostasis in the specific areas of neurological diseases. (edu.au)
  • Northbrook-based Marathon Pharmaceuticals has dropped out of the pharmaceutical industry's main lobbying group following controversy over the company's recent attempt to price a muscular dystrophy drug at $89,000 a year. (chicagotribune.com)
  • When Marathon Pharmaceuticals' $89,000 price tag for a year's supply of its newly approved muscular dystrophy drug sparked outrage earlier this month, the company's CEO responded with another figure: $20. (chicagotribune.com)
  • Marathon Pharmaceuticals' $89,000 price tag for a muscular dystrophy drug has drawn the ire of the pharmaceutical industry's main lobbying group, which is reconsidering whether it wants companies like Marathon as members. (chicagotribune.com)
  • Marathon Pharmaceuticals is only 6 years old, but this week's uproar surrounding its $89,000 muscular dystrophy drug isn't the first time the company - or its CEO - has faced outrage over drug pricing. (chicagotribune.com)
  • Learning how to direct the activity of Tregs has important implications for improving cancer immunotherapy as well as developing better treatments for autoimmune diseases such as rheumatoid arthritis and type 1 diabetes. (salk.edu)
  • The severity of this devastating disease makes it imperative to find effective treatments for which important progress has been made recently and will be discussed in this review. (nature.com)
  • I began writing about genetics decades ago, and the best thing about getting older is witnessing the development of targeted treatments for single-gene diseases that I never thought would happen. (plos.org)
  • The companies are primarily developing treatments for blindness, blood disorders, liver disease, and cancer. (technologyreview.com)
  • If the data is positive, the experimental treatment could be rapidly approved for use in humans with Duchenne because of cardiac stem cell treatments have been approved for other patient populations, including those with advanced heart disease. (bio-medicine.org)
  • Elizabeth Russell's work led to new treatments for blood and immunological diseases, including performing the first bone marrow transplant in a mammal. (jax.org)