Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD)
Goselink RJ, Schreuder TH, Mul K, Voermans NC, Pelsma M, de Groot IJ, van Alfen N, Franck B, Theelen T, Lemmers RJ, Mah JK, van der Maarel SM, van Engelen BG, Erasmus CE.
BMC Neurol. 2016. 17;16:138.
Protocol of a population-based prospective cohort study on Facio-scapulohumeral dystrophy in children. The children will be assessed at baseline and at 2-year follow-up. The primary outcome is the motor function as measured by the MFM.
Keywords: Facioscapulohumeral dystrophy; Muscular dystrophies; Neuromuscular diseases; Observational study; Pediatrics
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