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