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Clinical trial preparedness in facioscapulohumeral dystrophy: Outcome measures and patient access

✍ Scribed by Tawil, R.; Shaw, D.W.; van der Maarel, S.M.; Tapscott, S.J.

Book ID
123490303
Publisher
Elsevier Science
Year
2014
Tongue
English
Weight
233 KB
Volume
24
Category
Article
ISSN
0960-8966

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## Abstract We studied the reliability of a series of endpoints in an evaluation of subjects with Duchenne muscular dystrophy (DMD). The endpoints included quantitative muscle tests (QMTs), timed function tests, forced vital capacity (FVC), and manual muscle tests (MMT). Thirty‐one ambulatory subje