靶向 DMPK 的反义寡核苷酸在 1 型肌强直性营养不良患者中的应用：一项多中心、随机、剂量递增、安慰剂对照、Ⅰ/Ⅱa 期试验。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。Lancet Neurol。2023 年 3 月；22(3):218-228。doi: 10.1016/S1474-4422(23)00001-7。PMID: 36804094
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. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. PMID: 36804094