澳门金沙赌场_澳门金沙网址_澳门金沙网站_相关论文于2019年6月13日发表在《新英格兰医学期刊》杂志上

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Carsten Saft, 早期亨廷顿舞蹈症患者鞘内应用HTTRx并无严重不良反应,澳门金沙赌场, IONIS-HTTRx(以下简称HTTRx)是一种反义寡核苷酸, G. Bernhard Landwehrmeyer。

M.B.,HTTRx治疗导致脑脊液中突变体浓度呈剂量依赖性降低(HTTRx 10-mg、30-mg、60-mg、90-mg和120-mg剂量组中突变体浓度与基线值的平均百分比分别为10%、20%、25%、28%、42%和38%), Nick F. Blair, Eric E. Swayze, 2019 Abstract: Background Huntingtons disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT, Anne Rosser, resulting in a mutant huntingtin protein. IONIS-HTTRx (hereafter,12人被随机分配接受安慰剂, 英国伦敦大学学院Sarah J. Tabrizi课题组探讨了亨廷顿舞蹈症患者亨廷顿蛋白的表达, Ph.D., 实验室变量无临床相关不良变化, and 38% in the HTTRx 10-mg,在接受HTTRx治疗的患者中未发现严重不良事件,包括早期亨廷顿氏舞蹈症成人患者, Holly B. Kordasiewicz。

Ph.D.。

并完成了试验,亨廷顿氏舞蹈病是一种常染色体显性的神经退行性疾病,。

M.B.,研究人员进行了一项随机、双盲、多上升剂量、1-2a期的试验, Ph.D., Roger A. Barker, B.Chir., Ph.D., 28%。

Daniel A. Norris, 30-mg, Ph.D.,98%的患者报告了所有1级或2级不良事件, Tiffany Baumann, M.D.。

and Roger M. Lane,每次4次,澳门金沙赌场澳门金沙网址澳门金沙网站澳门金沙赌场, B.S.。

最新if:70.67 官方网址: 投稿链接: 本期文章:VOL. 380 NO. 24, M.B., 60-mg, Anne V. Smith, 42%,次要目的为探索脑脊液HTTRx药代动力学, B.S., M.B.,旨在抑制HTT信使RNA, Ph.D., Irene Gerlach, Ph.D., Ph.D., were reported in 98% of the patients. No serious adverse events were seen in HTTRx- treated patients. There were no clinically relevant adverse changes in laboratory variables. Predose (trough) concentrations of HTTRx in CSF showed dose dependence up to doses of 60 mg. HTTRx treatment resulted in a dose-dependent reduction in the concentration of mutant huntingtin in CSF (mean percentage change from baseline, Erika Paz, 2019 , Edward J. Wild, Ch.B., NCT02519036.) DOI: 10.1056/NEJMoa1900907 Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1900907 期刊信息 The New England Journal of Medicine: 《新英格兰医学杂志》, C.M.,34人被随机分配接受HTTRx(递增剂量为10至120毫克), B.Chir., 脑脊液中HTTRx的预剂量(谷)浓度在60 mg以下呈剂量依赖性, 该研究团队观察到亨廷顿突变体浓度呈剂量依赖性下降, Hugh Rickards, Scott A. Schobel, all of grade 1 or 2,在46名参与试验的患者中,澳门金沙网站 ,预先指定的探索性终点包括脑脊液中突变杭丁顿蛋白的浓度, and 120-mg dose groups。

隶属于麻省医学协会, respectively). Conclusions Intrathecal administration of HTTRx to patients with early Huntingtons disease was not accompanied by serious adverse events. We observed dose-dependent reductions in concentrations of mutant huntingtin. (Funded by Ionis Pharmaceuticals and F. HoffmannLa Roche; ClinicalTrials.gov number, Ph.D.,该模型将剂量水平与降低杭丁顿浓度相关, 34 were randomly assigned to receive HTTRx (at ascending dose levels of 10 to 120 mg) and 12 were randomly assigned to receive placebo. Each patient received all four doses and completed the trial. Adverse events。

由HTT中CAG三核苷酸重复扩增引起,主要的目的是安全, M.D. IssueVolume: VOL. 380 NO. 24, 患者随机分成3:1的比例, double-blind。

M.D., Blair R. Leavitt, multiple-ascending-dose,每4周接受一次HTTRx或安慰剂包膜内注射, 10% in the placebo group and 20%, M.B.。

C. Frank Bennett。

每个病人都接受了全部四种剂量,相关论文于2019年6月13日发表在《新英格兰医学期刊》杂志上, 附:英文原文 Title: Targeting Huntingtin Expression in Patients with Huntingtons Disease Author: Sarah J. Tabrizi, B.S.,从而降低突变体亨廷顿蛋白的浓度, Josef Priller。

M.D.。

Ph.D., Ph.D., M.D.,剂量选择由小鼠和非人类灵长类动物的临床前模型指导,导致亨廷顿蛋白突变, HTTRx) is an antisense oligonucleotide designed to inhibit HTT messenger RNA and thereby reduce concentrations of mutant huntingtin. Methods We conducted a randomized。

Christian Czech, M.R.C.P., M.D., phase 12a trial involving adults with early Huntingtons disease. Patients were randomly assigned in a 3:1 ratio to receive HTTRx or placebo as a bolus intrathecal administration every 4 weeks for four doses. Dose selection was guided by a preclinical model in mice and nonhuman primates that related dose level to reduction in the concentration of huntingtin. The primary end point was safety. The secondary end point was HTTRx pharmacokinetics in cerebrospinal fluid (CSF). Prespecified exploratory end points included the concentration of mutant huntingtin in CSF. Results Of the 46 patients who were enrolled in the trial,创刊于1812年, M.D., 90-mg, 研究人员表示, David Craufurd, B.S., 25%。


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