Hope is on the Horizon for Patients with Duchenne Muscular Dystrophy
As a genetic disorder characterized by progressive muscle degeneration and weakness, Duchenne muscular dystrophy, or DMD is a virulent form of muscular dystrophy. The absence of dystrophin, which is a...
View ArticleDrisapersen Attains Breakthrough Status
On June 27, 2013 GlaxoSmithKline announced that Drisapersen, a medication designed to treat Duchenne muscular dystrophy, had received breakthrough status form U.S. regulators. The designation of...
View ArticleDMD Treatments Eteplirsen and Drisapersen in the Spotlight
Recent doubt from the FDA regarding Eteplirsen has caused considerable concern for Sarepta Therapeutics Inc. The pending NDA for Eteplirsen has been effectively put on hold by the FDA, based on...
View ArticleFDA Offers Support for Use of Drisapersen
Drisapersen recently received a positive nod from the FDA. After several recent productive meetings with the FDA Prosensa announced that they will submit Drisapersen for an approval in 2014. Already...
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