An Antibody–Oligonucleotide Conjugate for Myotonic Dystrophy Type 1

20262 citationsJournal Article

Authors

Nicholas E. Johnson · Virginia Commonwealth University

Li-Jung Tai · Electronic BioSciences (United States)

Johanna Hamel · University of Rochester Medical Center

J Day · Stanford Medicine

Jeffrey M. Statland · University of Kansas Medical Center

Payam Soltanzadeh · University of California, Los Angeles

Sankarasubramoney H. Subramony

Abstract

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.).

Topics & Keywords

Genetic Neurodegenerative Diseases Autoimmune Neurological Disorders and Treatments Amyotrophic Lateral Sclerosis Research

UN Sustainable Development Goals

Zero hunger

Publication Details

Published in: New England Journal of Medicine

Volume 394, Issue 8, pp. 763-772

DOI: 10.1056/nejmoa2407326

Field-Weighted Citation Impact: 43.57