Sarep­ta gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy fails on func­tion — rais­ing se­ri­ous ques­tions about all their drugs

Sarep­ta $SRPT has some ex­plain­ing to do.

Af­ter the mar­ket closed on Thurs­day the con­tro­ver­sial biotech com­pa­ny re­port­ed that their next-gen gene ther­a­py ap­proach to Duchenne mus­cu­lar dy­s­tro­phy did what it was sup­posed to do in spurring a sig­nif­i­cant in­crease in dy­s­trophin pro­duc­tion, but failed on the key end­point that aimed to demon­strate im­proved mus­cle func­tion for the boys af­flict­ed with this lethal con­di­tion.

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