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This designation is pivotal for developing DYNE-251, which is currently being evaluated in the Phase I/II DELIVER trial for DMD individuals amenable to exon 51 skipping. The trial’s long-term ...
3d
Pharmaceutical Technology on MSNEC grants orphan drug designation to Dyne Therapeutics’ DMD therapyThe company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.
Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.
DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of ...
The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.
DYNE-251 is currently being assessed in the Phase 1/2 DELIVER global clinical trial, which targets individuals with DMD amenable to exon 51 skipping. Recent long-term clinical data from this trial ...
DYNE-251, currently in a Phase 1/2 clinical trial named DELIVER, is designed for patients with DMD amenable to exon 51 skipping. Recently, the trial showcased promising long-term results ...
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