Vyondys 53™ (golodirsen) – New orphan drug approval
December 12, 2019 - The FDA announced the approval of Sarepta Therapeutics’ Vyondys 53 (golodirsen), for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
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