Dyne announces new long-term data from DMD clinical trial skipping Exon 51

Exon 51

CureDuchenne provided early funding to Dyne Therapeutics, and is pleased to share that Dyne has released new long-term data from the DELIVER trial in individuals amenable to skipping Exon 51.  At the 6-month time point, individuals treated with 20 mg/kg of DYNE-251 every 4 weeks had a mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content).  Improvements in functional outcomes were also observed through 18 months, including improvements in Stride Velocity 95th Centile (SV95C) that exceeded the published proposed minimal clinically important difference (MCID).  Full data from the DELIVER trial is expected in late 2025, and Dyne plans on submitting a Biologics License Application (BLA) to the FDA for accelerated approval in early 2026.

Read the full press release:
https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-long-term-clinical-data-phase-12

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