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BioMarin Fails to Win Approval for Drug to Treat Duchenne

January 14, 2016

BioMarin Pharmaceutical Inc. failed to win U.S. approval for a drug to treat Duchenne muscular dystrophy, a deadly genetic disease that usually affects young boys.

The Food and Drug Administration rejected the once-a-week injection known as Kyndrisa, according to a statement from the company Thursday. A panel of outside advisers voted in November that the medication, known chemically as drisapersen, didn’t conclusively show improved walking ability in patients with the muscle-wasting disease.Click here for full article.