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Dyne Therapeutics Says Trial of Potential Duchenne Muscular Dystrophy Drug Shows 'Meaningful' Results
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Dyne Therapeutics Says Trial of Potential Duchenne Muscular Dystrophy Drug Shows 'Meaningful' Results
Mar 17, 2025 1:21 AM

04:11 AM EDT, 03/17/2025 (MT Newswires) -- Dyne Therapeutics ( DYN ) said Sunday that long-term clinical data from an ongoing phase 1/2 clinical trial of its investigational drug DYNE-251 is showing "clinically meaningful and sustained functional improvement" in patients with Duchenne muscular dystrophy.

The 32-patient trial is specifically for individuals with the rare genetic disease who are amenable to exon 41 skipping, a type of genetic therapy, the company said.

Improvements were seen for standardized assessments of stride velocity, movement, 10-meter walk or time, and time to rise from floor, the company said.

Dyne said it expects to submit a Biologics License Application submission for accelerated US approval in early 2026.

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