Effectiveness of Baliforsen, an Antisense Oligonucleotide Targeting DMPK, in Patients With Myotonic Dystrophy Type 1
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The Lancet Neurology
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Lancet Neurol 2023 Mar 01;22(3)218-228, CA Thornton, RT Moxley, K Eichinger, C Heatwole, L Mignon, WD Arnold, T Ashizawa, JW Day, G Dent, MK Tanner, T Duong, EP Greene, L Herbelin, NE Johnson, W King, JT Kissel, DG Leung, DJ Lott, DA Norris, EM Pucillo, W Schell, JM Statland, N Stinson, SH Subramony, S Xia, KM Bishop, CF BennettFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.