Bioethics News

After Eteplirsen: Duchenne Stakeholders Contemplate What Comes Next

(Chemical & Engineering News) – On Sept. 19, the Food & Drug Administration announced it had granted “accelerated approval,” a conditional stamp based on limited data, to Sarepta Therapeutics’ Duchenne muscular dystrophy treatment eteplirsen. The agency’s decision closed a tumultuous chapter for a rare disease community and opened a new one about how drugs should be tested and reviewed. Eteplirsen, the first drug for a deadly disease that affects children, was one of the most closely watched treatments in the pharmaceutical industry. It had also become one of the most contentious.

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