Dyne Therapeutics Says Trial of Potential Duchenne Muscular Dystrophy Drug Shows 'Meaningful' Results
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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