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sliceanddice59

11/30/17 9:44 PM

#132472 RE: Mikesc #132456

Yes. Remember that Sarepta's Duchenne Muscular Dystrophy trial consisted of 12 patients with no placebo arm. It was approved based on RWE and vocal parents. It seems that, for a rare disease like DMD, it's not "business as usual" for the FDA. And Rett's, like DMD, is a rare disease.