BREAKING NEWS: The FDA has 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.
We are filled with hope.
bit.ly/eteplirseninfo(from the Muscular Dystrophy Association)
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Sarepta is seeking accelerated approval for eteplirsen for patients with DMD who have a confirmed mutation of the dystrophin gene
amenable to exon 51 skipping (≈13% of patients with DMD).
In such patients, skipping of exon 51 might restore the reading frame of dystrophin, increase the production of dystrophin, and lead to
a clinical benefit for patients.
(from the FDA proposal, which you may see
here)