Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.

LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN.
BMC Neurol. 2019. 10;19(1):224.
The primary goal of this study is to hasten drug development for FSHD by validating two novel clinical outcome assessments (COAs). An 18-month longitudinal study in 220 genetically confirmed and clinically affected participants will be performed.
MFM-D1 will be used as an outcome measure.

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Keywords: Biomarkers; Clinical trial; Electrical impedance Myography; Facioscapulohumeral muscular dystrophy; Functional testing; Muscular dystrophy; Outcome measures.

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