The U.S. Food and Drug Administration granted an accelerated approval to Sarepta Therapeutics for its Vyondys 53 (golodirsen) injection for the treatment of a particular rare mutation in Duchenne muscular dystrophy (DMD) that is associated with 8% of DMD patients. The treatment, which works by skipping exon 53 of the dystrophin gene, is the first treatment to target this particular mutation. Yourway is encouraged by the increase of treatments for orphan diseases being granted accelerated approval designations, helping accelerate the process of getting badly needed drugs to patients with unmet needs.
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