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top123

02/14/20 12:24 PM

#236377 RE: TempePhil #236376

Temp - also from my notes not sure who post it but i will re post it and hope dont mind.. will go to gym now and have a good and bless day AVXL long.

" Sarepta's drug eteplirsen for Duchenne Muscular Dystrophy, another rare disease. The FDA approved this drug on the basis of a 12 patient trial with no placebo arm . However much of the approval had to do with RWE and a highly vocal group of parents."
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bennyboy1

02/14/20 12:44 PM

#236388 RE: TempePhil #236376

Absolutely, nothing much better than Real World Evidence! Seeing an afflicted person improve before your eyes - can almost say Miraculous!!!!!!!!!!!!!