Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Resource Type
Article
Authors
Thornton, Charles A ; Moxley, Richard Thomas, III ; Eichinger, Katy ; Heatwole, Chad ; Mignon, Laurence ; Arnold, W David ; Ashizawa, Tetsuo ; Day, John W ; Dent, Gersham ; Tanner, Matthew K ; Duong, Tina ; Greene, Ericka P ; Herbelin, Laura ; Johnson, Nicholas E ; King, Wendy ; Kissel, John T ; Leung, Doris G ; Lott, Donovan J ; Norris, Daniel A ; Pucillo, Evan M ; Schell, Wendy ; Statland, Jeffrey M ; Stinson, Nikia ; Subramony, Sub H ; Xia, Shuting ; Bishop, Kathie M ; Bennett, C Frank
Source
In The Lancet Neurology March 2023 22(3):218-228
Subject
Primary Research Articles
Language
ISSN
1474-4422