"The agency has just granted that use expansion, and it turns out that it was all due to Peter Marks, who completely overruled three review teams and two of his highest-level staffers (all of whom said that Sarepta had not proven its case)."
It's sad when drugs are sold on hope and not on science. This reminds me of Biogen's Alzheimer's drug that eventually was stopped after millions were spent by people hoping it would work with controversial clinical results: