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Duchenne Muscular Dystrophy


Eteplirsen CAS 1173755-55-9

The U.S. Food and Drug Administration approved Eteplirsen CAS# 1173755-55-9 (marketed as Exondys 51) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the[…]

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As a debilitating disease which destroys the muscles Duchenne Muscular Dystrophy, also known as DMD is a genetic disorder. DMD affects mainly males, and it is characterized by extreme muscle fatigue and excessive muscle degeneration. The cause of Duchenne Muscular Dystrophy is a lack of dystrophin in the body, which is the necessary protein that[…]

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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 concerns regarding Phase 2b study results. Patients suffering from Duchenne muscular dystrophy {DMD} are in dire need of effectual, safe and tolerable treatments to[…]

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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 “breakthrough” status is certainly noteworthy, as the FDA created this new status at the beginning of 2013 to indicate medications that proved to offer significant benefits over other medications[…]

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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 protein that helps to keep muscle cells intact, is the cause of this genetic condition. Duchenne muscular dystrophy is one of the nine types of[…]

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