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Sarepta Completes NDA for Eteplirsen and FDA Accepts BioMarin NDA for Drisapersen; Two DMD Treatments on Horizon

Background: On Monday, June 29, BioMarin announced  that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen. Both "exon-skipping" drugs target exon 51, and may help up to 13% of Duchenne muscular dystrophy patients.

Isis Pharmaceuticals Reports Data from ISIS-SMN Rx in Children with Type 2 and 3 SMA

News: Isis Pharmaceuticals Reports Data from Phase 2 Study in Infants with Spinal Muscular Atrophy

It's Myasthenia Gravis Awareness Month 2015

In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.

FDA issues draft guidance on developing drugs for Duchenne Muscular Dystrophy

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.

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