Gene Therapy for Kids’ Deadly Muscle Disease Fails to Reach Trial Goal

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Gene Therapy for Kids’ Deadly Muscle Disease Fails to Reach Trial Goal
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(Bloomberg) -- Roche Holding AG shares dropped after a trial of its gene therapy for Duchenne muscular dystrophy did not meet the main goal in a study, a...

-- Roche Holding AG shares dropped after a trial of its gene therapy for Duchenne muscular dystrophy did not meet the main goal in a study, a setback for the Swiss drugmaker and its partner Sarepta Therapeutics Inc.The stock fell as much as 2.9% in Zurich trading. Sarepta disclosed the findings late Monday, saying patients improved in an ambulatory assessment, yet the change wasn’t great enough to hit the clinical trial’s target.

The therapy secured accelerated US approval in June, a regulatory shortcut that allows companies to market medications before completing definitive trials in cases where patients have few or no treatment options. Sarepta on Monday said it plans to ask the FDA to expand the labeled indication to treat older patients. The stock was halted after US markets closed.

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