Novel image analysis to assess molecular functionality of dystrophin in Duchenne muscular dystrophy clinical trials

被引：0

作者：

Scaglioni, D. ^{[1
]}

Catapano, F. ^{[1
]}

Ellis, M. ^{[1
]}

Torelli, S. ^{[1
]}

Chambers, D. ^{[1
]}

Feng, L. ^{[1
]}

Husayni, S. ^{[2
]}

Malhotra, J. ^{[2
]}

Harriman, S. ^{[2
]}

Koenig, E. ^{[2
]}

Dugar, A. ^{[2
]}

Steiner, D. ^{[2
]}

Morgan, J. ^{[1
]}

Phadke, R. ^{[1
]}

Muntoni, F. ^{[1
]}

机构：

[1] UCL, London, England

[2] Sarepta Therapeut Inc, Cambridge, MA USA

来源：

NEUROMUSCULAR DISORDERS | 2020年 / 30卷

关键词：

D O I：

10.1016/j.nmd.2020.08.126

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.123

引用

页码：S82 / S82

页数：1

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[21] Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy [J].

Wells, Dominic J. .

JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY, 2006, 27 (5-7) :387-398

[22] CHARACTERIZATION OF DYSTROPHIN IN CARRIERS OF DUCHENNE MUSCULAR-DYSTROPHY [J].

CLERK, A ;

RODILLO, E ;

HECKMATT, JZ ;

DUBOWITZ, V ;

STRONG, PN ;

SEWRY, CA .

JOURNAL OF THE NEUROLOGICAL SCIENCES, 1991, 102 (02) :197-205

[23] Cognition Associated with Dystrophin Deletion in Duchenne Muscular Dystrophy [J].

Cho, Soo Kyoung ;

Moon, Jae Ho ;

Lee, Sang Chul ;

Yoo, Tae Won ;

Ha, Young Ran ;

Park, Young Bum ;

Choi, Won Ah .

ANNALS OF REHABILITATION MEDICINE-ARM, 2006, 30 (03) :225-229

[24] Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy [J].

Lake, Stephen L. ;

Quintana, Melanie A. ;

Broglio, Kristine ;

Panagoulias, Jennifer ;

Berry, Scott M. ;

Panzara, Michael A. .

STATISTICS IN MEDICINE, 2021, 40 (19) :4167-4184

[25] CLINICAL-TRIALS OF ALLOPURINOL IN DUCHENNE MUSCULAR-DYSTROPHY [J].

THOMSON, WHS .

MEDICAL HYPOTHESES, 1985, 17 (02) :175-189

[26] Monoclonal antibodies for clinical trials of Duchenne muscular dystrophy therapy [J].

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Nguyen Thi Man ;

Morris, Glenn E. .

NEUROMUSCULAR DISORDERS, 2014, 24 (03) :195-200

[27] CLINICAL TRIALS IN DUCHENNE MUSCULAR DYSTROPHY: WHERE ARE WE IN 2016? [J].

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[28] Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy [J].

Koo, Taeyoung ;

Wood, Matthew J. .

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[29] Bayesian Adaptive Design for Clinical Trials in Duchenne Muscular Dystrophy [J].

Lake, Stephen .

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[30] Micro-Dystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy [J].

Shin, Jin-Hong ;

Pan, Xiufang ;

Hakim, Chady H. ;

Yang, Hsiao T. ;

Yue, Yougping ;

Zhang, Keqing ;

Terjung, Ronald L. ;

Duan, Dongsheng .

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