Duchenne Muscular Dystrophy (DMD)

New Muscular Dystrophy Drugs Offer Hope

Moving Toward 'Next-Generation' Gene Therapy

Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).

DMD UPDATE: Drisapersen Outperforms Placebo in Walking Test

DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug

A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.

UPDATE: ACE-031 Clinical Trials in Duchenne MD

Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted.

‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research

Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24.

A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in clinical trials for neuromuscular diseases, and the unique opportunity the conference provided for information-sharing and collaboration among scientific professionals from many disciplines.

MDA Scientific Conference To Emphasize Therapy Development

The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.

DMD/BMD: MDA Funds Development of Drug to Fix Calcium Leaks

MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

DMD: FDA Asks Sarepta for More Data on Eteplirsen

The U.S. Food and Drug Administration (FDA) has said it will consider an application for accelerated approval for eteplirsen, an experimental drug for Duchenne muscular dystrophy (DMD), after it reviews additional data from clinical trials of the drug.

DMD: Drisapersen Outperforms Placebo in Walking Test

The exon-skipping drug drisapersen has shown encouraging results in an international phase 2b trial in boys with Duchenne muscular dystrophy (DMD) caused by specific mutations.

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