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

Heart-break­ing 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 …

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