scholarly journals Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings

2011 ◽  
Vol 1 (9) ◽  
pp. 1217-1235 ◽  
Author(s):  
Kate Bushby ◽  
Edward Connor
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Working Group ◽  
International Working Group ◽  
Group Meetings

S.P.21 Clinical outcome measures in Collagen 6 (COL6) and Laminin α2(LAMA2) related congenital muscular dystrophy

2012 ◽  
Vol 22 (9-10) ◽  
pp. 893
Author(s):  
K.G. Meilleur ◽  
M. Jain ◽  
E. Kim ◽  
L. Hynan ◽  
C.Y. Shieh ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Congenital Muscular Dystrophy ◽  
Laminin Α2

Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy

2019 ◽  
Vol 61 (1) ◽  
pp. 63-68 ◽  
Author(s):  
Sara Nagy ◽  
Sabine Schädelin ◽  
Patricia Hafner ◽  
Ulrike Bonati ◽  
Delia Scherrer ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures

P16 Towards a consensus on biochemical outcome measures for Duchenne muscular dystrophy clinical trials

2014 ◽  
Vol 24 ◽  
pp. S11
Author(s):  
S. Torelli ◽  
K. Anthony ◽  
V. Arechavala-Gomeza ◽  
L.E. Taylor ◽  
A. Vulin ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures

P1.50 Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy

2011 ◽  
Vol 21 (9-10) ◽  
pp. 656
Author(s):  
F. Pinto Mariz ◽  
I. Barthélémy ◽  
E. Yada ◽  
T. Voit ◽  
S.D. Silva-Barbosa ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Clinical Outcome ◽  
Predictive Markers ◽  
Dog Model

PO195 Outcome measures for duchenne muscular dystrophy: implications for trials

2017 ◽  
Vol 88 (Suppl 1) ◽  
pp. A63.1-A63
Author(s):  
Joana Domingos ◽  
Michelle Eagle ◽  
Amelie Moraux ◽  
Jordan Butler ◽  
Valerie Decostre ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures

EP.95Patient perception of outcome measures for non-ambulant Duchenne muscular dystrophy patients

2019 ◽  
Vol 29 ◽  
pp. S180
Author(s):  
K. Naarding ◽  
M. Van der Holst ◽  
N. Van de Velde ◽  
P. Van Schaik-Bank ◽  
H. Kan ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Outcome Measures
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