Sarepta’s Next-Gen DMD Drug SRP-5051 Shows Advantages, But Safety Concerns Too

The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope
Sarepta announced data on four patients from the first part of its Phase II trial of SRP-5051 • Source: Shutterstock

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