Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability.
A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are able to travel to Los Angeles.
The Muscular Dystrophy Association has awarded 44 new grants totaling $13.6 million to advance the understanding and treatment of neuromuscular diseases. The new grants, most of which took effect Feb. 1, encompass a range of diseases covered by MDA’s research program, and they support innovative approaches to basic research and new drug development.
A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.
For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.
In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin and how these interactions differ in
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 w
In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for Duchenne muscular dystroph
Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the
The drug tadalafil (Cialis, Adcirca), which dilates blood vessels and is approved to treat erectile dysfunction and pulmonary hypertension, has been found to improve blood flow to exercising forearm muscles in people with Becker muscular dystrophy (BMD).
The multinational pharmaceutical company GlaxoSmithKline (GSK) has announced promising results for its phase 1 trial of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy (DMD) who are no longer walking.
Experimental anti-cancer drugs that block IAP (inhibitor of apoptosis) genes and kill cancer cells have unexpectedly been found to induce the growth and repair of muscle tissue, say scientists supported in part by MDA.
A left ventricular assist device (LVAD), which helps the heart pump blood throughout the body, was implanted in the chest of a young man with Duchenne muscular dystrophy (DMD) at Cincinnati Children's Hospital Medical Center in September.
A multicenter study of the use of magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure the progression of Duchenne muscular dystrophy (DMD) is open at sites in Florida, Oregon and Pennsylvania for boys with DMD who meet study criteria.