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In Focus

Voice of the People

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When patients demand more than science can provide, high-priced, ineffective treatments can reach the market.

On April 25, 2016, a procession of doctors, researchers, parents and adolescent boys—many of the last group in wheelchairs—approached a microphone, one after the other, and asked a Food and Drug Administration advisory committee to recommend approval of an experimental drug called eteplirsen. It’s not unusual for several clinicians and patients to be i...

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