US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

Eteplirsen Granted Accelerated Approval!


A New Drug & New Hope for Kids with Duchenne

For decades we've funded foundational Duchenne muscular dystrophy (DMD) research. Twenty years ago, we supported the early-stage development of a new drug called eteplirsen (brand name Exondys 51). Today, we celebrate accelerated approval by the FDA!

This advancement could not have been made possible without our supporters and donors. Thank you to everyone who walked, ran and fundraised for the cause, to everyone who dropped a dollar in a fireman’s boot, texted in a telethon donation or raised awareness on social media. You have made this milestone possible!

  • 5 years ago

    There were no approved therapies to treat the underlying cause of DMD
  • 1 year ago

    Sarepta Therapeutics announced plans to submit a New Drug Application for eteplirsen
  • September 19, 2016

    Eteplirsen is granted accelerated approval to treat some forms of DMD

And this is just the beginning

Seeing this treatment become an available option for our families is a significant step forward - one that we know will spark more innovation and drug development across diseases. There are many other promising therapies moving closer to the finish line, but we can't keep this momentum going without you.