Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production.
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.
Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration has granted Fast Track designation to the company's most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.
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.
Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children.
A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve blood flow in the muscles of boys with Duchenne muscular dystrophy, according to a study published in the May 7, 2014, online issue of Neurology®, the medical journal of the American Academy of Neurology.
Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).
Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today reported financial results for the three months and year ended December 31, 2013, and provided an update of recent corporate developments.
Researchers at Washington University School of Medicine in St. Louis have demonstrated a new approach to treating muscular dystrophy. Mice with a form of this muscle-weakening disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste.
Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.
The Muscular Dystrophy Association has awarded research grants totaling over $1,000,000 to three Penn Medicine researchers: James Shorter, PhD, associate professor of Biochemistry and Biophysics; Hansell Stedman, MD, associate professor of Surgery; and Lee Sweeney, PhD, director of the Center for Orphan Disease Research and Therapy.
A preclinical study led by researchers at Children's National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy.
New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease.
Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health.
A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle.
Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.
Newport Beach-based nonprofit Coalition Duchenne has awarded a $150,000 grant to a Cedars-Sinai Heart Institute team investigating whether an experimental cardiac stem cell treatment could be used to treat Duchenne muscular dystrophy patients who have developed heart disease.
ARMGO Pharma and the Muscular Dystrophy Association today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug application with the U.S. Food and Drug Administration for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy.
DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.
Researchers at the University of Minnesota's Lillehei Heart Institute have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne Muscular Dystrophy (DMD).