, Columnist
Duchenne Race May Have No Winner
String of FDA disappointments suggests tougher road for orphan drugs.
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The worst kind of race is one that everyone loses.
Since their peaks last year, BioMarin, Sarepta, and PTC Therapeutics have fallen 49.32 percent, 63.68 percent, and 85.72 percent, respectively. The three companies are trying to get the first drug for Duchenne Muscular Dystrophy (DMD), a deadly and incurable form of M.D. that affects young boys, approved in the U.S.