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Let’s Make Duchenne History

Duchenne muscular dystrophy is the #1 genetic killer of children worldwide. We’re making historic progress to beat it.

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Eteplirsen Approved

9.19.16

Majorly HUGE historic moment!!! The FDA finally approved Eteplirsen. We’ve supported this therapeutic approach since 2005, and for the past three years we’ve been fighting for its approval. You helped make this happen. We need more medicines to help more kids, but for today be proud. Be happy. Be invigorated. We are making a difference.

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We are Charley’s Fund

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DEPLOYED to advance new therapies

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Charley

Charley, a soon-to-be 16 year old spitfire, is our original inspiration. Over the past 12 years we have met many other children whose colorful personalities and intense passions deepen our resolve to end Duchenne.

Him

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Updates & Inspiration

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Homepage NewsNewsResearch News
September 19, 2016

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys…
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