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Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study

Abstract:

The field of translational research in Duchenne muscular dystrophy (DMD) has been transformed in the last decade by a number of therapeutic targets, mostly studied in ambulant patients. A paucity of studies focus on measures that capture the non-ambulant stage of the disease, and the transition between the ambulant and non-ambulant phase. In this prospective natural history study, we report the results of a comprehensive assessment of respiratory, upper limb function and upper limb muscle str...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1016/j.nmd.2019.02.002

Authors


Publisher:
Elsevier
Journal:
Neuromuscular Disorders More from this journal
Volume:
29
Issue:
4
Pages:
261-268
Publication date:
2019-02-19
Acceptance date:
2020-02-08
DOI:
EISSN:
1873-2364
ISSN:
0960-8966
Pmid:
30852071
Language:
English
Keywords:
Pubs id:
1092860
Local pid:
pubs:1092860
Deposit date:
2020-03-12

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