FDA ad­comm sup­ports first po­ten­tial drug for ul­tra-rare, fa­tal Barth syn­drome

Heart­break­ing anec­dotes from the pa­tients, par­ents and doc­tors of chil­dren with the ul­tra-rare and fa­tal Barth syn­drome won over an FDA ad­vi­so­ry com­mit­tee on Thurs­day …

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER