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

共 50 条

[1] Clinical outcome study for dysferlinopathy: three years of natural history data for clinical trial readiness
James, M.
Mayhew, A.
Lofra, R. Muni
Jacobs, M.
Canal, A.
Duong, T.
Gee, R.
Harman, M.
Holsten, S.
Lowes, L.
Maron, E.
Mendez, B.
Pedrosa Belmonte, I.
Sakamoto, C.
Semplicini, C.
Siener, C.
Thiele, S.
Vandervelde, B.
Bushby, K.
Straub, V.
NEUROMUSCULAR DISORDERS, 2018, 28 : S33 - S34
[2] The international clinical outcome study for dysferlinopathy - ten years of natural history data
James, M.
Dressman, H. Gordish
Rufibach, L.
Hilsden, H.
Mayhew, A.
Straub, V.
NEUROMUSCULAR DISORDERS, 2023, 33 : S166 - S166
[3] The clinical outcome study for dysferlinopathy: pregnancy in dysferlinopathy
Moore, U.
James, M.
Jacobs, M.
Mayhew, A.
Spuler, S.
Day, J.
Bharucha-Goebel, D.
Stojkovic, T.
Mendell, J.
Straub, V.
NEUROMUSCULAR DISORDERS, 2019, 29 : S104 - S104
[4] Is cardiac dysfunction a feature of dysferlinopathy? Data from the clinical outcome study of dysferlinopathy
Torron, R. Fernandez
Harris, E.
Bourke, J.
Bettinson, K.
Hilsden, H.
Spuler, S.
Day, J.
Jones, K.
Bharucha-Goebel, D.
Salort-Campana, E.
Pestronk, A.
Walter, M.
Paradas, C.
Stojkovic, T.
Mori-Yoshimura, M.
Bravver, E.
Diaz-Manera, J.
Pegoraro, E.
Mendell, J.
Bushby, K.
Straub, V.
NEUROMUSCULAR DISORDERS, 2017, 27 : S145 - S145
[5] Is cardiac dysfunction a feature of dysferlinopathy? Data from the Clinical Outcome Study of Dysferlinopathy
Tiet, M.
Fernandez-Torron, R.
Bourke, J.
Bettinson, K.
Harris, E.
Hilsden, H.
Spuler, S.
Day, J. W.
Jones, K. J.
Bharucha-Goebel, D. X.
Salort-Campana, E.
Pestronk, A.
Walter, M. C.
Paradas, C.
Stojkovic, T.
Mori-Yoshimura, M.
Bravver, E.
Manera, J. Diaz
Pegoraro, E.
Mendell, J. R.
Bushby, K.
Straub, V.
NEUROMUSCULAR DISORDERS, 2018, 28 : S5 - S6
[6] THE INTERNATIONAL CLINICAL OUTCOME STUDY IN DYSFERLINOPATHY
Ghaoui, Roula
Meredith, James
Fernandez-Torron, Roberto
Moore, Ursula
Diaz-Manera, Jordi
Smith, Fiona E.
Mayhew, Anna
Jones, Kristi J.
Bushby, Kate
Straub, Volker
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2017, 88 (05):
[7] The International Dysferlinopathy Registry and an international clinical outcome study for dysferlinopathy
von Rekowski, B.
Blandin, G.
Krahn, M.
Beroud, C.
De Waele, L.
Bushby, K.
NEUROMUSCULAR DISORDERS, 2012, 22 : S35 - S35
[8] Clinical Outcome Study of Dysferlinopathy: what are the best outcome measures for dysferlinopathy patients?
James, M. K.
Jacobs, M.
Mayhew, A.
Feng, J.
Spuler, S.
Day, J. W.
Jones, K. J.
Bharucha-Goebel, D. X.
Salort-Campana, E.
Pestronk, A.
Walter, M. C.
Paradas, C.
Stojkovic, T.
Mori-Yoshimura, M.
Bravver, E.
Manera, J. Diaz
Pegoraro, E.
Mendell, J. R.
Bushby, K.
Straub, V.
NEUROMUSCULAR DISORDERS, 2018, 28 : S6 - S6
[9] Clinical outcome study of dysferlinopathy: what are the best outcome measures for dysferlinopathy patients?
James, M.
Jacobs, M.
Mayhew, A.
Feng, J.
Spuler, S.
Day, J.
Jones, K.
Bharucha-Goebel, D.
Salort-Campana, E.
Pestronk, A.
Walter, M.
Paradas, C.
Stojkovic, T.
Mori-Yoshimura, M.
Bravver, E.
Diaz-Manera, J.
Pegoraro, E.
Mendell, J.
Bushby, K.
Straub, V.
NEUROMUSCULAR DISORDERS, 2017, 27 : S227 - S227
[10] Quality of life in adults with dysferlinopathy: international clinical outcome study of dysferlinopathy
Hilsden, H.
James, M.
Dressman, H. Gordish
Day, J.
Mendell, J.
Torron, R. Fernandez
Harms, M.
Pestronk, A.
Vissing, J.
Desai, U.
Yoshimura, M.
Shin, J.
Mozaffar, T.
Stojkovic, T.
Pegoraro, E.
Raivas, J. Bevilacqu
Olive, M.
Paradas, C.
Straub, V.
Mayhew, A.
NEUROMUSCULAR DISORDERS, 2023, 33 : S113 - S113

← 1 2 3 4 5 →