scholarly journals Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment

2021 ◽  
Author(s):  
Craig M. McDonald ◽  
Lee‐Jen Wei ◽  
Kevin M. Flanigan ◽  
Gary Elfring ◽  
Panayiota Trifillis ◽  
...  
Keyword(s):  
Ambulatory Assessment ◽  
The North

M.P.2.01 Reliability of the north star ambulatory assessment in a multicentric setting

2009 ◽  
Vol 19 (8-9) ◽  
pp. 571
Author(s):  
E.S. Mazzone ◽  
S. Messina ◽  
G. Vasco ◽  
M. Main ◽  
M. Eagle ◽  
...  
Keyword(s):  
Ambulatory Assessment ◽  
The North

Reliability and validity analyses of the North Star Ambulatory Assessment in Brazilian Portuguese

2017 ◽  
Vol 27 (8) ◽  
pp. 723-729 ◽  
Author(s):  
Larissa O. Okama ◽  
Lívia M. Zampieri ◽  
Carina L. Ramos ◽  
Flávia O. Toledo ◽  
Cyntia R.J. Alves ◽  
...  
Keyword(s):  
Reliability And Validity ◽  
Brazilian Portuguese ◽  
Ambulatory Assessment ◽  
The North

S.P.5 Trial readiness: Clinical interpretability of change scores of the North Star Ambulatory Assessment in Duchenne muscular dystropy

2012 ◽  
Vol 22 (9-10) ◽  
pp. 876-877
Author(s):  
A.G. Mayhew ◽  
M. Eagle ◽  
E. Scott ◽  
K.M.D. Bushby ◽  
M. Adnan ◽  
...  
Keyword(s):  
Ambulatory Assessment ◽  
Change Scores ◽  
The North ◽  
Duchenne Muscular Dystropy

scholarly journals North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

PLoS ONE ◽  
2021 ◽  
Vol 16 (6) ◽  
pp. e0253882
Author(s):  
Giorgia Coratti ◽  
Marika Pane ◽  
Claudia Brogna ◽  
Valeria Ricotti ◽  
Sonia Messina ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Ambulatory Assessment ◽  
The North ◽  
Lower Baseline ◽  
Single Deletion ◽  
Progression Of Disease ◽  
Baseline Values ◽  
The Difference

Introduction The aim of this study was to report 36-month longitudinal changes using the North Star Ambulatory Assessment (NSAA) in ambulant patients affected by Duchenne muscular dystrophy amenable to skip exons 44, 45, 51 or 53. Materials and methods We included 101 patients, 34 had deletions amenable to skip exon 44, 25 exon 45, 19 exon 51, and 28 exon 53, not recruited in any ongoing clinical trials. Five patients were counted to skip exon 51 and 53 since they had a single deletion of exon 52. Results The difference between subgroups (skip 44, 45, 51 and 53) was significant at 12 (p = 0.043), 24 (p = 0.005) and 36 months (p≤0.001). Discussion Mutations amenable to skip exons 53 and 51 had lower baseline values and more negative changes than the other subgroups while those amenable to skip exon 44 had higher scores both at baseline and at follow up. Conclusion Our results confirm different progression of disease in subgroups of patients with deletions amenable to skip different exons. This information is relevant as current long term clinical trials are using the NSAA in these subgroups of mutations.

scholarly journals Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

PLoS ONE ◽  
2019 ◽  
Vol 14 (9) ◽  
pp. e0221097 ◽  
Author(s):  
Francesco Muntoni ◽  
Joana Domingos ◽  
Adnan Y. Manzur ◽  
Anna Mayhew ◽  
Michela Guglieri ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Ambulatory Assessment ◽  
Individual Item ◽  
The North

Reliability of the North Star Ambulatory Assessment in a multicentric setting

2009 ◽  
Vol 19 (7) ◽  
pp. 458-461 ◽  
Author(s):  
E.S. Mazzone ◽  
S. Messina ◽  
G. Vasco ◽  
M. Main ◽  
M. Eagle ◽  
...  
Keyword(s):  
Ambulatory Assessment ◽  
The North

scholarly journals Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy

2013 ◽  
Vol 55 (11) ◽  
pp. 1046-1052 ◽  
Author(s):  
Anna G Mayhew ◽  
Stefan J Cano ◽  
Elaine Scott ◽  
Michelle Eagle ◽  
Kate Bushby ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Ambulatory Assessment ◽  
Meaningful Change ◽  
The North
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