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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial LANCET NEUROLOGY Thornton, C. A., Iii, R., Eichinger, K., Heatwole, C., Mignon, L., Arnold, W., Ashizawa, T., Day, J. W., Dent, G., Tanner, M. K., Duong, T., Greene, E. P., Herbelin, L., Johnson, N. E., King, W., Kissel, J., Leung, D. G., Lott, D. J., Norris, D. A., Pucillo, E. M., Schell, W., Statland, J. M., Stinson, N., Subramony, S. H., Xia, S., Bishop, K. M., Bennett, C. 2023; 22 (3): 218-228View details for Web of Science ID 000996507100001