CLINICAL OUTCOME STUDY FOR DYSFERLINOPATHY: THREE YEARS OF NATURAL HISTORY DATA FOR CLINICAL TRIAL READINESS

被引：0

作者：

James, M. K. ^{[1
]}

Mayhew, A. G. ^{[1
]}

Jacobs, M. ^{[2
,3
]}

Eagle, M.

Maron, E. ^{[4
]}

Gee, R. ^{[5
]}

Harman, M. ^{[6
]}

Duong, T. ^{[7
]}

Vandervelde, B. ^{[8
]}

Siener, C. ^{[9
]}

Thiele, S. ^{[10
]}

Mendez, B. ^{[11
]}

Canal, A. ^{[12
]}

Sakamoto, C. ^{[13
]}

Holsten, S. ^{[14
]}

Pedrosa Belmonte, I. ^{[15
]}

Semplicini, C. ^{[16
]}

Alfano, L. ^{[17
]}

Lowes, L. ^{[17
]}

Straub, V. ^{[1
]}

机构：

[1] John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, Tyne & Wear, England

[2] Childrens Natl Hlth Syst, Ctr Translat Sci, Div Biostat & Study Methodol, Washington, DC USA

[3] George Washington Univ, Pediat Epidemiol & Biostat, Washington, DC USA

[4] Elan Physio, Berlin, Germany

[5] Stanford Univ, Sch Med, Dept Neurol & Neurol Sci, Stanford, CA 94305 USA

[6] Childrens Hosp Westmead, Inst Neurosci & Muscle Res, Sydney, NSW, Australia

[7] Childrens Natl Hlth Syst, Dept Neurol, Washington, DC USA

[8] Aix Marseille Univ, La Timone Hosp, Neuromuscular & ALS Ctr, Marseille, France

[9] Washington Univ, Sch Med, Dept Neurol, St Louis, MO USA

[10] Ludwig Maximilians Univ Munchen, Friedrich Baur Inst, Dept Neurol, Munich, Germany

[11] Hosp Univ Virgen Rocio, Inst Biomed Sevilla, Dept Neurol, Neuromuscular Unit, Seville, Spain

[12] AP HP, Inst Myol, GH Pitie Salpe Triere 47-83,Blvd Hop, Paris, France

[13] Natl Ctr Neurol & Psychiat Tokyo, Natl Ctr Hosp, Dept Neurol, Tokyo, Japan

[14] Carolinas Healthcare Syst Neurosci Inst, Charlotte, NC USA

[15] Hosp Santa Creu & Sant Pau, Neurol Dept, Neuromuscular Disorders Unit, Barcelona, Spain

[16] Univ Padua, Dept Neurosci, Padua, Italy

[17] Nationwide Childrens Hosp, Columbus, OH USA

来源：

MUSCLE & NERVE | 2018年 / 58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：

引用

页码：S3 / S3

页数：1

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