P.150Consistency of 48-week changes in North Star Ambulatory Assessment (NSAA) between Duchenne muscular dystrophy natural history data and clinical trial placebo arms, after adjustment for prognostic factors

2019 ◽  
Vol 29 ◽  
pp. S92
Author(s):  
N. Goemans ◽  
B. Wong ◽  
F. Muntoni ◽  
C. McDonald ◽  
E. Mercuri ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Prognostic Factors ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Ambulatory Assessment ◽  
History Data

scholarly journals Correction: Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

PLoS ONE ◽  
2019 ◽  
Vol 14 (7) ◽  
pp. e0220714
Author(s):  
Claudia Brogna ◽  
Giorgia Coratt ◽  
Marika Pane ◽  
Valeria Ricotti ◽  
Sonia Messina ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data

scholarly journals Correction: Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes

PLoS ONE ◽  
2015 ◽  
Vol 10 (12) ◽  
pp. e0144079 ◽  
Author(s):  
Marika Pane ◽  
Elena Stacy Mazzone ◽  
Serena Sivo ◽  
Maria Pia Sormani ◽  
Sonia Messina ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data

P.148Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

2019 ◽  
Vol 29 ◽  
pp. S91
Author(s):  
C. Brogna ◽  
G. Coratti ◽  
M. Pane ◽  
V. Ricotti ◽  
S. Messina ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data

scholarly journals PRO125 SUITABILITY OF NATURAL HISTORY DATA FOR EXTERNAL CONTROLS IN DUCHENNE MUSCULAR DYSTROPHY

2019 ◽  
Vol 22 ◽  
pp. S864
Author(s):  
N. Goemans ◽  
B. Wong ◽  
F. Muntoni ◽  
C. McDonald ◽  
E. Mercuri ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data

scholarly journals Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

PLoS ONE ◽  
2019 ◽  
Vol 14 (6) ◽  
pp. e0218683 ◽  
Author(s):  
Claudia Brogna ◽  
Giorgia Coratti ◽  
Marika Pane ◽  
Valeria Ricotti ◽  
Sonia Messina ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data

scholarly journals Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes

PLoS ONE ◽  
2014 ◽  
Vol 9 (10) ◽  
pp. e108205 ◽  
Author(s):  
Marika Pane ◽  
Elena Stacy Mazzone ◽  
Serena Sivo ◽  
Maria Pia Sormani ◽  
Sonia Messina ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
History Data
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