Eteplirsen Granted Accelerated Approval!
CLICK TO PLAYA 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!
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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
Articles & Stories
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Read MoreEteplirsen Granted Accelerated Approval to Treat DMD
On Sept. 19, 2016, the FDA granted accelerated approval to eteplirsen for the treatment of Duchenne muscular dystrophy! Decades of MDA research and investment have paid off with the first disease-modifying drug available to treat DMD.
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Read MoreA Letter About the Eteplirsen Advisory Committee Outcome
A recap of our time at the FDA advisory committee meeting where we stood shoulder to shoulder with our sister groups, research and clinical experts, and most importantly, our families.
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Read MoreMDA's Testimony at FDA Advisory Committee Hearing
Watch the video of Dr. Valerie Cwik's speech at the FDA's Advisory Committee hearing where she gives her review of eteplirsen.
