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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...
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DMD 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...
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Ataluren Phase 3 Data Encouraging for Cystic Fibrosis
Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic...
View ArticleHope 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 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 ArticleAtaluren Phase 3 Data Encouraging for Cystic Fibrosis
Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic...
View Article
