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.
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 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.
Watch the video of Dr. Valerie Cwik's speech at the FDA's Advisory Committee hearing where she gives her review of eteplirsen.
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.