Safety and Efficacy of DYNE-101 in Adults with DM1: Phase 1/2 ACHIEVE Trial Data (S16.003)
Determine the safety and efficacy of DYNE-101 in adults with myotonic dystrophy type 1 (DM1) in the Phase 1/2 ACHIEVE trial (NCT05481879).
Authors
- Joost Kools (ORCID: https://orcid.org/0000-0002-6838-9345)
- Douglas A. Kerr
- Valeria Sansone (ORCID: https://orcid.org/0000-0002-1354-3800)
- James B Lilleker (ORCID: https://orcid.org/0000-0002-9230-4137)
- Guillaume Bassez (ORCID: https://orcid.org/0000-0002-2044-1052)
- S. K. Ray (ORCID: https://orcid.org/0000-0002-8099-6690)
- Christopher G. Turner (ORCID: https://orcid.org/0000-0002-3371-9260)
- Bing Han (ORCID: https://orcid.org/0000-0001-9763-0372)
- Richard H Roxburgh (ORCID: https://orcid.org/0000-0002-8930-7003)
- Benedikt G. H. Schoser (ORCID: https://orcid.org/0000-0002-2757-8131)
- Chris Mix
- Daniel Wolf (ORCID: https://orcid.org/0000-0001-5000-8578)
- Marika Pane (ORCID: https://orcid.org/0000-0002-4851-6124)
- Jordi Díaz‐Manera (ORCID: https://orcid.org/0000-0003-2941-7988)
Institutions
- Radboud University Nijmegen (NL)
- University of Auckland (NZ)
- University of Milan (IT)
- Manchester Academic Health Science Centre (GB)
- Agostino Gemelli University Polyclinic (IT)
- University Medical Center (US)
- Radboud University Medical Center (NL)
- Northern Health and Social Care Trust (GB)
- Centro Clinico Nemo (IT)
- Institut de Myologie (FR)
- Friedrich Baur Stiftung (DE)
- University College London (GB)
- Newcastle University (GB)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000210508
- Citations
- 3
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 5.45