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Home » Sarepta Reports Promising Results for Its Experimental Gene Therapy Targeting Duchenne Muscular Dystrophy

Sarepta Reports Promising Results for Its Experimental Gene Therapy Targeting Duchenne Muscular Dystrophy

January 12, 2022

Sarepta Therapeutics announced positive results from a phase 2 study on SRP-9001 (delandistrogene moxeparvovec), its experimental gene therapy for Duchenne muscular dystrophy, a progressive and deadly genetic disease with no cure.

Sarepta compared trial volunteers who were switched from placebo to treatment to patients in a separate control group, matched by age and disease severity. Among the treated patients, Sarepta reported an average 1.3-point increase on a standard test of motor function, versus a projected 0.7-point decline for the control group.

The improvement was observed in children who were just over 7 years old at the start of the trial.

Results seen across multiple trials “bolsters our confidence in the potential disease-modifying benefits of this therapy and reinforces our conviction in the probability of success of EMBARK, our large, phase 3 placebo-controlled global study presently underway and dosing,” said Sarepta’s CEO, Doug Ingram.

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