Dyne Therapeutics (DYN, Financial) has announced a significant milestone with the European Commission granting orphan drug designation to its investigational therapy, DYNE-251, for the treatment of Duchenne muscular dystrophy (DMD).
DYNE-251 is currently under evaluation in the Phase 1/2 DELIVER global clinical trial, targeting patients who can benefit from exon 51 skipping. The trial is generating promising results, with long-term data revealing significant and ongoing functional improvements at the designated dose, as presented in March at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.
The DELIVER trial's functional assessments include Stride Velocity 95th Centile, a digitally measured outcome recognized as a primary endpoint for DMD clinical trials in Europe. This development marks a crucial step forward in advancing treatment options for individuals with DMD.
