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# Dyne Files BLA for Duchenne Muscular Dystrophy Therapy
- URL: https://www.fdaweb.com/dyne-files-bla-for-duchenne-muscular-dystrophy-therapy/
- Published: 2026-05-26T12:00:00.000Z
- Updated: 2026-09-14T13:39:51.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5161201

Dyne Therapeutics has submitted a BLA that seeks accelerated approval for its experimental Duchenne muscular dystrophy (DMD) therapy z-rostudirsen for patients amenable to exon 51 skipping.

The submission is based on data from the registrational expansion cohort of the Phase 1/2 DELIVER study, in which the therapy demonstrated a statistically significant increase in dystrophin production, the company says. Dyne also reports functional improvements across multiple clinical endpoints along with what it describes as a favorable safety profile.

The investigational therapy, also known as DYNE-251, is designed for patients with mutations in the DMD gene that can be treated through exon 51 skipping. Dyne is proposing a dosing regimen of 20 mg/kg administered intravenously once every four weeks.

The drug uses a phosphorodiamidate morpholino oligomer linked to an antibody fragment targeting transferrin receptor 1, a design intended to improve delivery into muscle tissue and the central nervous system, according to the company. The therapy is intended to restore production of near-full-length dystrophin, the protein missing in patients with DMD.