Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
Discover Now: Mutations and Deletions Amenable to Exon 51 Skipping Therapies
Results of DYNE-251 Clinical Trial
- Registrational Expansion Cohort (REC) met primary endpoint, demonstrating statistically significant increase in dystrophin to 5.46% at 6 months (muscle content-adjusted; p<0.0001), replicating the same 7-fold change from baseline previously observed at the registrational dose –
- Functional improvement was observed across multiple clinical endpoints at 6 months in REC; lung function was preserved at 6 months –
- New positive long-term results from DELIVER trial showed sustained functional improvement across all assessed endpoints through 24 months –
- Continued favorable safety and tolerability profile –
- Submission for U.S. Accelerated Approval on track for Q2 2026 –
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My son is suffering from dmd exon 49-50