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Researchers review ten years of scientific studies on mitochondrial toxicity in pregnant women

Researchers review ten years of scientific studies on mitochondrial toxicity in pregnant women

Researchers from the Hospital Clinic of Barcelona (Spain) have reviewed ten years' worth of scientific studies on mitochondrial toxicity in pregnant women. Exposure to toxic agents such as viruses, certain drugs, pesticides, alcohol and tobacco cause mitochondrial diseases about which very little is known, and which are transmitted from the mother to the foetus. [More]
EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

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]
Bern researchers develop active substance for treating Duchenne muscular dystrophy

Bern researchers develop active substance for treating Duchenne muscular dystrophy

Duchenne muscular dystrophy is a congenital disease which causes muscle degeneration and eventual death in teenagers. Recently, researchers from Bern developed an active substance, which they together with an international team tested successfully. [More]
PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC begins rolling NDA submission for Translarna to treat nmDMD

PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application to the United States Food and Drug Administration for Translarnaâ„¢ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). [More]
Researchers restore missing repair protein in skeletal muscle of patients with muscular dystrophy

Researchers restore missing repair protein in skeletal muscle of patients with muscular dystrophy

Advances in the treatment of muscular dystrophy: For the first time, a research team has succeeded in restoring a missing repair protein in skeletal muscle of patients with muscular dystrophy. [More]
Idera collaborates with TMA to advance new potential treatment approach for myositis

Idera collaborates with TMA to advance new potential treatment approach for myositis

Idera Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing nucleic acid therapeutics for patients with cancer and rare diseases, and The Myositis Association (TMA), the only nonprofit organization dedicated to solely serving all patients with inflammatory myopathies, today announced a collaboration to advance a new potential treatment approach for polymyositis and dermatomyositis known as Toll-like receptor (TLR) antagonism. [More]
UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

Millions of people in the United States have a circulatory problem of the legs called peripheral vascular disease. It can be painful and may even require surgery in serious cases. This disease can lead to severe skeletal muscle wasting and, in turn, limb amputation. [More]
PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics, Inc. today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna (ataluren) available to patients before commercial availability in certain countries. [More]
EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. [More]
Researcher identifies ''conductor'' in development of muscle tissue

Researcher identifies ''conductor'' in development of muscle tissue

A team led by Jean-Fran-ois C-t-, researcher at the IRCM, identified a ''conductor'' in the development of muscle tissue. The discovery, published online yesterday by the scientific journal Proceedings of the National Academy of Sciences (PNAS), could have an important impact on the treatment of muscular diseases such as myopathies and muscular dystrophies. [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]
Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

​Researchers at the Kennedy Krieger Institute recently announced study findings showing the successful development of a humanized preclinical model for facioscapulohumeral muscular dystrophy (FSHD), providing scientists with a much needed tool to accelerate novel therapeutic research and development. [More]
Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy. [More]
Scientists find key gene that activates muscle growth

Scientists find key gene that activates muscle growth

For decades, scientists have searched for treatments for myopathies - genetic muscular diseases such as muscular dystrophy and ALS, also called Lou Gehrig's disease. [More]
Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA). [More]
New insights into filaminopathies

New insights into filaminopathies

New insights into certain muscle diseases, the filaminopathies, are reported by an international research team led by Dr. Rudolf Andre Kley of the RUB's University Hospital Bergmannsheil in the journal Brain. [More]
Statin's potency may cause muscle problems in patients with cardiovascular disease

Statin's potency may cause muscle problems in patients with cardiovascular disease

A study from the University of California, San Diego School of Medicine, published August 22 online by PLoS ONE, reports that muscle problems reported by patients taking statins were related to the strength or potency of the given cholesterol-lowering drugs. [More]
MitoTarget Project and neurodegenerative diseases: an interview with Rebecca Pruss, Ph.D., CSO of Trophos

MitoTarget Project and neurodegenerative diseases: an interview with Rebecca Pruss, Ph.D., CSO of Trophos

The MitoTarget Project was a project funded by the European Commission, under their FP7 programme. It was funding for a call published in 2007. [More]
Researchers discover new cause of congenital myopathy

Researchers discover new cause of congenital myopathy

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics. [More]
Study identifies how skeletal muscle stem cells respond to muscle injury

Study identifies how skeletal muscle stem cells respond to muscle injury

A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure. [More]