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GSK muscular dystrophy drug gets "breakthrough" status
June 27, 2013 / 3:21 PM / 4 years ago

GSK muscular dystrophy drug gets "breakthrough" status

LONDON, June 27 (Reuters) - GlaxoSmithKline said on Thursday U.S. regulators have given breakthrough therapy status for its investigational compound drisapersen for the potential treatment of patients with duchenne muscular dystrophy (DMD).

The U.S. Food and Drug Administration (FDA) created the “breakthrough therapy” designation earlier this year for medicines deemed likely to demonstrate “substantial improvement” over existing drugs.

DMD is a debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births, GSK said, adding that the disease was caused by mutations in the dystrophin gene.

GSK said its clinical development plan evaluates the effect of drisapersen in ambulant (Phases II and III) and non-ambulant boys (Phase I) with DMD who have certain dystrophin gene mutations.

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