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Validation of motor and functional scales for the evaluation of adult patients with 5q spinal muscular atrophy
Objective To assess in adult spinal muscular atrophy (SMA) patients the construct validity and responsiveness of several outcome measures. Methods Patients older than 15 years and followed-up at least for 6 months, between October 2015 and August 2020, with one motor function scale (Hammersmith Functional Motor Scale Expanded, HFMSE; Revised Upper Limb module, RULM) in five referral centers were included. Bedside functional scales (Egen Klassification, EK2; Revised Amyotrophic Lateral Sclerosisdoi:10.1101/2021.06.12.21258357 fatcat:ds2ch5z66zfablpat4vkam3qjm