Pfizer Inc. has allowed a peek into its Phase Ib clinical data on PF-06939926, its investigational gene therapy for Duchenne muscular dystrophy (DMD), and while it does show some signs of efficacy, safety is a concern.
The primary endpoint of the ongoing Phase Ib study is to assess the safety and tolerability of the gene therapy, and one of the six boys participating had a severe immune reaction to the
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