Some families of the dozen boys involved in the clinical trial leading to the approval of Exondys, or eteplirsen, attest to the drug's effectiveness

Controversial drug approval stirs deep concerns — and hope

By American Chemical Society —— Bio and Archives--November 19, 2016

Health and Medicine | Comments | Back To Full Article

In September, the Food and Drug Administration approved Exondys, a controversial treatment for Duchenne muscular dystrophy based on tenuous data from just 12 patients. The cover story in Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society, explores what the decision could mean for future drugs for Duchenne and other rare diseases.

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