Eteplirsen Granted Accelerated Approval!CLICK TO PLAY
Your story matters
We hope this news is as exciting to you as it is to us. If you'd like to share your story with eteplirsen or thoughts on how this will impact our community, we'd love to hear it.
Articles & Stories
Eteplirsen 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.
A 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.
MDA'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.
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.