Posts By: carolinn

Next Thursday, an FDA advisory committee will meet to consider whether to approve a treatment for Duchenne muscular dystrophy. The last time an FDA advisory committee met to review a therapy for this fatal childhood disease, things did not go smoothly. — Portrayed in the media as a heated showdown between the FDA and desperate… Read more »

Sarepta Therapeutics announced this morning that the FDA has indicated it will not reach a final decision on eteplirsen by the May 26th deadline.  We understand and share the frustration many of you feel that the FDA still hasn’t made a final decision on eteplirsen despite strong scientific evidence that supports approval. We have already… Read more »

By next Thursday, the FDA will make a decision on approval for eteplirsen. Whether the FDA’s answer on eteplirsen is “YES,”or “YES, BUT…,” or “NO,” the race is far from over. If eteplirsen is approved, we will celebrate this historic milestone of the first ever therapy for DMD. For the 87% who cannot benefit from… Read more »

It’s not emotion, it’s science. We thank the scientific community from around the globe who came to Washington DC to defend the science behind eteplirsen. We encourage the @US_FDA to honor FDASIA, and fully utilize the tools Congress has given them to allow us to have this drug. ‪#‎MakeDuchenneHistory‬