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DYNE-251 in Duchenne muscular dystrophy amenable to exon 51 skipping
2023-510351-31-00Phase 2Authorised
- Sponsor
- Dyne Therapeutics
- Start
- Jan 2023
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 9
01
Overview
Phase 2, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing DYNE-251 multiple ascending dose with Placebo (2 arms). Primary endpoints: Treatment-emergent adverse events and Change in dystrophin protein levels at Week 25. Planned start Jan 2023; status: authorised.
02
Drugs
zeleciment rostudirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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