Gene-correction therapy suffers setback but is still seen as promising

09/25/2013 | New York Times (tiered subscription model), The

Anecdotal reports from patients and data from small clinical trials have raised hopes that experimental drugs for Duchenne muscular dystrophy based on a new genetic technique called exon skipping could ultimately lead to a treatment. Drisapersen, an exon-skipping drug being developed by GlaxoSmithKline and Prosensa, failed to beat a placebo in preserving muscle function in boys with the disease in a large trial, but the idea of correcting faulty genes remains promising. "We are fully confident in the exon-skipping technology as a viable platform to develop a treatment for Duchenne," said Debra Miller, president of the advocacy group CureDuchenne.

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