Facioscapulohumeral muscular dystrophy functional composite outcome measure.

Katy EichingerChad HeatwoleStanley IyaduraiWendy KingLindsay BakerSusanne HeiningerAmy BartlettNuran DilekWilliam B MartensMichael McdermottJohn T KisselRabi TawilJeffrey M Statland

Published in: Muscle & nerve (2018)

The FSHD-COM is a disease-relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test-retest reliability and cross-sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD-COM. Muscle Nerve, 2018.

Keyphrases

muscular dystrophy
clinical trial
cross sectional
current status
duchenne muscular dystrophy
randomized controlled trial
peripheral nerve
phase iii