“This is a really exciting time for MDA and the families that MDA serves. This is the first treatment that’s being approved by FDA for therapy for Duchenne Dystrophy. That’s a great thing, and it’s great for the families — they’re going to be able to have a drug that’s going to change the course of this disease.”

https://strongly.mda.org/eteplirsen-granted-accelerated-approval-to-treat-dmd/