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The myopathies are neuromuscular disorders in which the primary symptom is muscle weakness due to dysfunction of muscle fiber. Other symptoms of myopathy can include include muscle cramps, stiffness, and spasm. Myopathies can be inherited (such as the muscular dystrophies) or acquired (such as common muscle cramps).
aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today that Resolaris has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of facioscapulohumeral muscular dystrophy (FSHD). [More]
Joseph Roche receives grant to develop improved exercise programs for people with dysferlinopathies

Joseph Roche receives grant to develop improved exercise programs for people with dysferlinopathies

The Wayne State University Eugene Applebaum College of Pharmacy and Health Sciences today announced that Joseph Roche, assistant professor for the physical therapy program in the Department of Health Care Sciences, received a one-year, $100,000 grant from the Jain Foundation for "Developing a Multi-Pronged Strategy for the Clinical Management of Dysferlinopathies," a project that seeks to develop better exercise programs for people with dysferlinopathies, or dysferlin-linked muscular dystrophies. [More]
TGen scientists discover the likely cause of rare type of muscle weakness in six children

TGen scientists discover the likely cause of rare type of muscle weakness in six children

Scientists at the Translational Genomics Research Institute (TGen), using state-of-the-art genetic technology, have discovered the likely cause of a child's rare type of severe muscle weakness. [More]

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD). [More]
Clementia secures additional $10 million to support development of palovarotene for treatment of FOP

Clementia secures additional $10 million to support development of palovarotene for treatment of FOP

Clementia Pharmaceuticals, Inc. today announced that it has secured an additional $10 million from current investors to support development of the company's lead compound palovarotene for the treatment of fibrodysplasia ossificans progressive (FOP). [More]
AutoGenomics, Genomas sign agreement to develop DNA-guided diagnostic systems

AutoGenomics, Genomas sign agreement to develop DNA-guided diagnostic systems

AutoGenomics, Inc, a leader in automated microarray technology for molecular diagnostics, and Genomas, a biomedical company enabling DNA-guided medicine for personal healthcare, announced today that they have signed an agreement to jointly develop genetic tests and DNA-guided diagnostic systems for optimal selection of statins and for improved delivery of statin therapy for the treatment of cardiovascular disease, obesity and diabetes. [More]
Merck enrolls first patient in reformulated raltegravir Phase 3 trial for treatment of HIV-1 infection

Merck enrolls first patient in reformulated raltegravir Phase 3 trial for treatment of HIV-1 infection

Merck, known as MSD outside the United States and Canada, today announced that the first patient has been enrolled in the company's global Phase 3 clinical trial, ONCEMRK. [More]
Researchers show that vitamin B3 can slow down progression of mitochondrial disease

Researchers show that vitamin B3 can slow down progression of mitochondrial disease

The researchers of the University of Helsinki, Finland, and école Polytechnique Fédérale de Lausanne, Switzerland, have shown that vitamin B3 form nicotinamide riboside can slow down the progression of mitochondrial disease, suggesting its potential as a novel therapy approach to adult-onset mitochondrial muscle diseases. [More]
Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

The Jain Foundation is delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (COS), exceeding the original goal of 150 patients. [More]
Investigators urge drug regulators to provide clear evidence on statins’ side effects

Investigators urge drug regulators to provide clear evidence on statins’ side effects

At a time when the wider prescription of statins is under renewed public scrutiny, a substantial analysis of placebo-controlled randomised trials of statins has found that only a small minority of side effects reported by those taking the cholesterol-lowering drugs are actually attributable to them. [More]
Patients with congenital disorders are urged to register with CMDIR to advance clinical trials

Patients with congenital disorders are urged to register with CMDIR to advance clinical trials

If you are affected by Malignant Hyperthermia, Congenital Muscular Dystrophies or Congenital Myopathy Subtypes, the Malignant Hyperthermia Association of the United States and the Cure Congenital Muscle Disease organization are asking for your help to enable research and clinical trials by registering with the Congenital Muscle Disease International Registry (CMDIR). [More]
Study: Genetic mutations in titin gene can cause skeletal muscle disease

Study: Genetic mutations in titin gene can cause skeletal muscle disease

A University of Arizona doctoral candidate has shown for the first time that genetic mutations in the titin gene can cause skeletal muscle myopathy, a disease in which muscle fibers do not function properly, resulting in muscle weakness. [More]
Merck announces FDA approval of ISENTRESS for oral suspension

Merck announces FDA approval of ISENTRESS for oral suspension

Merck, known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration recently approved ISENTRESS for oral suspension, a new pediatric formulation of Merck's integrase inhibitor. With this approval, ISENTRESS is now indicated in combination with other antiretroviral agents for the treatment of HIV-1 infection in patients four weeks of age and older. [More]
New gene mutation helps doctors give more accurate diagnosis of mitochondrial myopathy

New gene mutation helps doctors give more accurate diagnosis of mitochondrial myopathy

A new gene mutation which will help doctors give a more accurate diagnosis of a particular type of brain and muscle disease in children has been discovered for the first time by University of Leeds experts. [More]
Soligenix initiates orBec Phase 2 study for GI manifestations of chronic GVHD

Soligenix initiates orBec Phase 2 study for GI manifestations of chronic GVHD

Soligenix, Inc., a clinical stage biopharmaceutical company focused on developing products to treat serious inflammatory diseases where there remains an unmet medical need, as well as developing several biodefense vaccines and therapeutics, announced today that it has initiated a Phase 2, randomized, double-blind, placebo-controlled study evaluating orBec (oral beclomethasone 17,21-dipropionate or BDP) as a treatment for the gastrointestinal manifestations of chronic Graft-versus-Host disease. [More]
Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

... [More]
Merck to present pharmacokinetic data on once-daily ISENTRESS at EACS

Merck to present pharmacokinetic data on once-daily ISENTRESS at EACS

Merck, known as MSD outside the United States and Canada, is presenting pharmacokinetic data this week on investigational formulations of a once daily dose of ISENTRESS at the 14th European AIDS Conference, sponsored by the European AIDS Clinical Society. [More]

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

... [More]
Four new pre-clinical drug development projects at NIH develop treatment for rare disease

Four new pre-clinical drug development projects at NIH develop treatment for rare disease

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers. [More]

Evotec And Jain Extend Research Collab In Skeletal Muscular Dystrophy

... [More]
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