A series of aggregated randomized-controlled N-of-1 trials with mexiletine in non-dystrophic myotonia: clinical trial results and validation of rare disease design

被引：0

作者：

Stunnenberg, Bas ^{[1
]}

Raaphorst, Joost ^{[1
]}

Groenewoud, Hans ^{[1
]}

Statland, Jeffrey ^{[2
]}

Griggs, Robert ^{[3
]}

Woertman, Willem ^{[1
]}

Stegeman, Dick ^{[1
]}

Timmermans, Janneke ^{[1
]}

Trivedi, Jaya ^{[4
]}

Matthews, Emma ^{[5
]}

Saris, Christiaan ^{[1
]}

Schouwenberg, Bas ^{[1
]}

Drost, Gea ^{[6
]}

van Engelen, Baziel ^{[1
]}

van der Wilt, GertJan ^{[1
]}

机构：

[1] Radboud Univ, Med Ctr, Nijmegen, Netherlands

[2] Univ Kansas, Med Ctr, Kansas City, KS 66103 USA

[3] Univ Rochester, Med Ctr, Rochester, NY 14642 USA

[4] UT Southwestern Med Ctr, Dallas, TX USA

[5] MRC Ctr Neuromuscular Dis, London, England

[6] Univ Med Ctr Groningen, Groningen, Netherlands

来源：

NEUROLOGY | 2018年 / 90卷

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P3.440

引用

页数：3

共 10 条

[1] COMBINED N-OF-1 TRIALS MEXILETINE VS. PLACEBO IN NON-DYSTROPHIC MYOTONIA USING A BAYESIAN APPROACH
Stunnenberg, B. C.
Woertman, W.
Raaphorst, J.
Timmermans, J.
van Alfen, N.
Schouwenberg, B.
Groenewoud, H.
van Engelen, B. G. M.
Drost, G.
van der Wilt, G. J.
MUSCLE & NERVE, 2014, 50 : S3 - S4
[2] Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol
Bas C Stunnenberg
Willem Woertman
Joost Raaphorst
Jeffrey M Statland
Robert C Griggs
Janneke Timmermans
Christiaan G Saris
Bas J Schouwenberg
Hans M Groenewoud
Dick F Stegeman
Baziel G M van Engelen
Gea Drost
Gert Jan van der Wilt
BMC Neurology, 15
[3] Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol
Stunnenberg, Bas C.
Woertman, Willem
Raaphorst, Joost
Statland, Jeffrey M.
Griggs, Robert C.
Timmermans, Janneke
Saris, Christiaan G.
Schouwenberg, Bas J.
Groenewoud, Hans M.
Stegeman, Dick F.
van Engelen, Baziel G. M.
Drost, Gea
van der Wilt, Gert Jan
BMC NEUROLOGY, 2015, 15
[4] Efficacy of mexiletine in non-dystrophic myotonia: results of an international multi-centred randomised controlled trial
Rayan, D. L. Raja
Matthews, E.
Barreto, G.
Tan, S. V.
Dewar, L.
Burge, J.
Wang, Y.
Trivedi, J.
Ciafaloni, E.
Salajegheh, M.
Venance, S.
Meola, G.
Bundy, B.
Herbelin, L.
Statland, J.
Griggs, R.
Barohn, R.
Hanna, M. G.
NEUROMUSCULAR DISORDERS, 2012, 22 : S4 - S4
[5] CLINICAL USEFULNESS OF AMITRIPTYLINE IN FIBROMYALGIA - THE RESULTS OF 23 N-OF-1 RANDOMIZED CONTROLLED TRIALS
JAESCHKE, R
ADACHI, J
GUYATT, G
KELLER, J
WONG, B
JOURNAL OF RHEUMATOLOGY, 1991, 18 (03) : 447 - 451
[6] Precision Medicine and Noradrenergic Biomarkers in PTSD: Preliminary Methods and Validation Data for an Aggregated N-Of-1 Clinical Trial Design Using Pupillometery and Cardiovascular Biomarkers
Hendrickson, Rebecca
Millard, Steve
Jane, Shofer
Schork, Nicholas
Raskind, Murray
NEUROPSYCHOPHARMACOLOGY, 2018, 43 : S230 - S230
[7] A series of N-of-1 trials to assess the therapeutic interchangeability of two enalapril formulations in the treatment of hypertension in Addis Ababa, Ethiopia: study protocol for a randomized controlled trial
Alemayehu, Chalachew
Mitchell, Geoffrey
Aseffa, Abraham
Clavarino, Alexandra
McGree, James
Nikles, Jane
TRIALS, 2017, 18
[8] A series of N-of-1 trials to assess the therapeutic interchangeability of two enalapril formulations in the treatment of hypertension in Addis Ababa, Ethiopia: study protocol for a randomized controlled trial
Chalachew Alemayehu
Geoffrey Mitchell
Abraham Aseffa
Alexandra Clavarino
James McGree
Jane Nikles
Trials, 18
[9] Validation Study of Kim's Sham Needle by Measuring Facial Temperature: An N-of-1 Randomized Double-Blind Placebo-Controlled Clinical Trial
Lee, Sanghun
Lim, Nara
Choi, Sunmi
Kim, Sungchul
EVIDENCE-BASED COMPLEMENTARY AND ALTERNATIVE MEDICINE, 2012, 2012
[10] Efficacy and Safety of Ginkgo Biloba Pills for Coronary Heart Disease with Impaired Glucose Regulation: Study Protocol for a Series of N-of-1 Randomized, Double-Blind, Placebo-Controlled Trials
Sun, Mingyue
Chai, Lulu
Lu, Fang
Zhao, Yang
Li, Qingna
Cui, Boya
Gao, Rui
Liu, Yue
EVIDENCE-BASED COMPLEMENTARY AND ALTERNATIVE MEDICINE, 2018, 2018

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