Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

Charles A. Thornton, Richard T. Moxley, Katy Eichinger, Chad Heatwole, Laurence Mignon, W. David Arnold, Tetsuo Ashizawa, John W Day, Gersham Dent, Matthew Tanner, Tina Duong, Ericka Greene, Laura Herbelin, Nicholas E. Johnson, Wendy King, John T. Kissel, Doris G. Leung, Donovan J. Lott, Daniel A Norris, Evan M Pucillo, W Schell, Jeffrey Statland, Nikia Stinson, S. H. Subramony, Shuting Xia, Kathie M. Bishop, C. Frank Bennett