research

FDA OKs Testing of OPMD Drug in US

BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).

DMD: New Strategy Aims to Change Dystrophin DNA

MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb.

Drug Development for DMD: February 2015 Update

Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.

Fighting fibrosis

Genervon's 'Master Regulator' Drug Encourages ALS Community

Biopharmaceutical company Genervon has announced what it considers encouraging  results from a "compassionate use" trial of its experimental drug GM604 in a single patient with advanced  amyotrophic lateral sclerosis (ALS).

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.

Stanford Biobank Seeks Tissue Samples

Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

Drug Development for DMD: Fall 2014 Update-Part 2

Several experimental drugs are i development to treat Duchenne muscular dystrophy (DMD), a genetic disorder that results in a lack of the dystrophin protein in cardiac and skeletal muscle cells.

BMD: Follistatin Gene Transfer Results Encouraging

Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

Drug Development for DMD: Fall 2014 Update

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

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