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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.
Thornton CA, Moxley RT 3rd, Eichinger K, Heatwole C, Mignon L, Arnold WD, Ashizawa T, Day JW, Dent G, Tanner MK, Duong T, Greene EP, Herbelin L, Johnson NE, King W, Kissel JT, Leung DG, Lott DJ, Norris DA, Pucillo EM, Schell W, Statland JM, Stinson N, Subramony SH, Xia S, Bishop KM, Bennett CF. Thornton CA, et al. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. Lancet Neurol. 2023. PMID: 36804094 Clinical Trial.
Reliability testing of the quantitative myasthenia gravis score.
Barohn RJ, McIntire D, Herbelin L, Wolfe GI, Nations S, Bryan WW. Barohn RJ, et al. Ann N Y Acad Sci. 1998 May 13;841:769-72. doi: 10.1111/j.1749-6632.1998.tb11015.x. Ann N Y Acad Sci. 1998. PMID: 9668327 No abstract available.
Myasthenia gravis activities of daily living profile.
Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Wolfe GI, et al. Neurology. 1999 Apr 22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. Neurology. 1999. PMID: 10227640
71 results