Muscular Dystrophies

Research and clinical trial updates

posted on October 8, 2015 - 9:23am
Towards Treatments and Cures New grants pave the way to progress

Research and clinical trial updates

posted on July 14, 2015 - 3:03pm
Quest Summer 2015
Powerful Research MDA welcomes a neurological disease expert to the family

Research and clinical trial updates

posted on April 21, 2015 - 9:03am
Quest Spring 2015
Research Leadership MDA welcomes two new muscle disease and ALS experts As part of MDA’s bold plan to accelerate treatments and cures for neuromuscular diseases, two new scientific program officers have joined the MDA family: Amanda Haidet-Phillips, Ph.D., and Laura Hagerty, Ph.D. Under the leadership of MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D., they will...

Four of six men with Becker muscular dystrophy increased their six-minute walking distance after injections of follistatin genes into the thigh muscles of both legs

posted on November 19, 2014 - 1:05pm
Update (March 24, 2015): Neurologist Jerry Mendell discusses the implications of this research in an approximately 20-minute podcast, part of the Nationwide Children's Hospital This Month in Muscular Dystrophy series.

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

posted on November 5, 2014 - 4:03pm
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014. PTC is moving forward with ataluren

Research updates and clinical trials information

posted on October 30, 2014 - 9:01am
Quest Fall 2014
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

Funding from MDA and three other organizations will allow ReveraGen BioPharma to test its experimental anti-inflammatory drug for Duchenne MD in healthy volunteers

posted on July 17, 2014 - 8:00am
ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.

Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations

posted on July 10, 2014 - 1:59pm
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.