An Antibody–Oligonucleotide Conjugate for Myotonic Dystrophy Type 1
Our results are consistent with delivery of del-desiran to muscle and amelioration of aberrant alternative splicing in some patients with myotonic dystrophy type 1; two serious adverse events occurred. These data support further clinical investigation. (Funded by Avidity Biosciences; ClinicalTrials.gov number, NCT05027269.).
Authors
- Bradley McEvoy
- Arthur A. Levin (ORCID: https://orcid.org/0000-0002-0949-2844)
- Payam Soltanzadeh (ORCID: https://orcid.org/0000-0001-5788-8619)
- Charles A. Thornton (ORCID: https://orcid.org/0000-0002-2124-6080)
- Jeanne Dekdebrun
- Johanna Hamel (ORCID: https://orcid.org/0000-0002-4851-7637)
- Amit Goel (ORCID: https://orcid.org/0000-0003-3525-9381)
- Steven G. Hughes
- Elizabeth Ackermann (ORCID: https://orcid.org/0000-0002-3714-2550)
- Sankarasubramoney H. Subramony
- Li-Jung Tai
- Miriam Freimer
- J Day
- Yiming Zhu
- W. David Arnold
- Matthew Wicklund
- Chao-Yin Chen
- Jeffrey M. Statland
- Kate Eichinger
- Nicholas E. Johnson
Institutions
- University of California, Los Angeles (US)
- Virginia Commonwealth University (US)
- University of Rochester Medical Center (US)
- University of Florida (US)
- Electronic BioSciences (United States) (US)
- University of Kansas Medical Center (US)
- Stanford Medicine (US)
- The University of Texas at San Antonio (US)
- The Ohio State University (US)
- University of Missouri (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2026-02-18
- DOI
- https://doi.org/10.1056/nejmoa2407326
- Citations
- 2
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 46.41