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."