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In a clinical trial earlier this year, an experimental gene therapy for Duchenne muscular dystrophy, licensed to Sarepta Therapeutics (SRPT), produced large increases in a crucial muscle protein normally missing in patients diagnosed with the disease.

Those results, disclosed in June, were better than expected but still left an important question unanswered: Would making more of this protein lead to meaningful improvements in muscle strength, movement, and function for Duchenne patients?

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