Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...