19 September 2016 - The experimental drug that the FDA approved Monday will only be used by a few thousand patients.
The approval may have set a precedent that could rocket through the health care system, opening the door for drug makers to get more medicines to market — even with scant evidence that they work.
The FDA’s decision elated families struggling with Duchenne muscular dystrophy, a rare and deadly disease. It sent the stock of the drug maker, Sarepta Therapeutics, soaring. It also touched off a barbed debate between those who applauded the move as giving hope to desperate patients — and those who warned it would backfire since there is no clear evidence the drug works.