Duchenne Muscular Dystrophy (DMD)

DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

Cambridge, Mass.-based biotechnology company Sarepta Therapeutics updated the Duchenne muscular dystrophy (DMD) community about its pipeline of drugs in development for this disorder via an Aug.

First Drug for Duchenne Muscular Dystrophy Gets 'Conditional Approval' in Europe

DMD: Ataluren Receives Conditional Approval in Europe

Update (Oct. 24, 2014): Enrollment for the phase 3 trial of ataluren is complete, with results expected in the second half of 2015. See the PTC press release of Sept. 9, 2014.

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original story:

PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ for the treatment of Nonsense Mutation Duchenne Muscular Dystrophy

DMD: ReveraGen Drug Will Move to Human Testing

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.

International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug

DMD: Eteplirsen Data Still Encouraging at 144 Weeks

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.

Sarepta Therapeutics Reports Long-Term Outcomes from Phase IIb Study of Eteplirsen

DMD: HT-100 Trial Has Reopened

Update (July 3, 2014): Akashi says the HT-100 main trial is now closed; the extension study is open to those who have already participated in the main trial.

Prosensa Will Pursue Accelerated Approval for Drisapersen to Treat DMD

Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.

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