What harm would be done, if at the end of the day the drug turned out to be a very powerful placebo? The boys who were on the trial had positive results and there were no adverse side effects. Because the patients do not have time on their side, the decision on whether or not to use the drug, should be placed with the patients and their doctors.
It seems to me that certain members of the FDA must lack empathy.
Sarepta Rare Disease Advisory Panel Meeting today, page-9
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