FDA Grants Accelerated Approval to First Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation

The FDA has granted accelerated approval to golodirsen injection (Vyondys 53, Sarepta Therapeutics) to treat patients with Duchenne muscular dystrophy (DMD) who have confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

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