In a Phase 3 trial, Pfizer’s gene therapy for Duchenne muscular dystrophy did not slow the disease’s progression, marking the second failed attempt in the field. The study, involving 99 boys aged 4-7, did not show any positive results on key secondary endpoints. This is similar to a previous trial of a Sarepta gene therapy that also failed to meet primary endpoints but showed some effect on secondary measures. Despite these setbacks, research in gene therapy for Duchenne muscular dystrophy continues, highlighting the challenges and complexities of treating this rare and devastating condition.
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Pfizer’s gene therapy for Duchenne muscular dystrophy fails trial
