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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. Among authors: johnson ne. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. Lancet Neurol. 2023. PMID: 36804094 Clinical Trial.
Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1).
Heatwole C, Bode R, Johnson N, Quinn C, Martens W, McDermott MP, Rothrock N, Thornton C, Vickrey B, Victorson D, Moxley R 3rd. Heatwole C, et al. Neurology. 2012 Jul 24;79(4):348-57. doi: 10.1212/WNL.0b013e318260cbe6. Epub 2012 Jul 11. Neurology. 2012. PMID: 22786587 Free PMC article.
Myotonic dystrophy: from bench to bedside.
Johnson NE, Heatwole CR. Johnson NE, et al. Semin Neurol. 2012 Jul;32(3):246-54. doi: 10.1055/s-0032-1329202. Epub 2012 Nov 1. Semin Neurol. 2012. PMID: 23117949 Review.
193 results