Rare Disease Treatment Unexpectedly Rejected by the FDA

September 4, 2019

Rare Disease Treatment Unexpectedly Rejected by the FDA

In a surprise decision, the U.S. Food and Drug Administration has rejected Sarepta’s experimental exon-skipping drug Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy, citing risks of both infection and kidney toxicity. Yourway is dedicated to supporting clinical trials for advanced therapies for orphan diseases.

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