Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Titel:
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Auteur:
Thornton, Charles A Moxley III, Richard Thomas 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