Safety and Efficacy of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (S31.010)
Determine the safety and efficacy of DYNE-251 in the Phase 1/2 DELIVER trial (NCT05524883) of males aged 4–16 with DMD mutations amenable to exon 51 skipping.
Authors
- Maria L. Naylor (ORCID: https://orcid.org/0000-0003-3844-6035)
- Michela Guglieri (ORCID: https://orcid.org/0000-0002-8455-0637)
- Soma Ray
- A. Dugar
- Liesbeth De Waele (ORCID: https://orcid.org/0000-0001-7126-575X)
- Chris Mix
- Kevin M. Flanigan (ORCID: https://orcid.org/0000-0001-6440-3376)
- Dajiang Wang (ORCID: https://orcid.org/0000-0002-8726-1996)
- Michelle Lorentzos
- Perry B. Shieh (ORCID: https://orcid.org/0000-0001-7145-7663)
- Douglas S. Kerr
- Craig Campbell
- H. Phan
- Nicolas Deconink
Institutions
- University of California, Los Angeles (US)
- Royal Victoria Infirmary (GB)
- London Health Sciences Centre (CA)
- Children's Hospital at Westmead (AU)
- Rare Disease Therapeutics (United States) (US)
- Newcastle University (GB)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211198
- Primary Topic
- Chemical Reactions and Isotopes
- Type
- article
- Field-Weighted Citation Impact
- 0.00