Rasch analysis to evaluate the motor function measure for patients with facioscapulohumeral muscular dystrophy

Mul K, Horlings CGC, Faber CG, van Engelen BGM, Merkies ISJ.
Int J Rehabil Res. 2021. 1;44(1):38-44.
A Rasch analyses was performed on MFM data from 194 FSHD patients to assess clinimetric properties in this patient group.
Rasch analysis revealed multiple limitations of the MFM for FSHD, the most important being a large ceiling effect and suboptimal sample-item targeting, which were most pronounced for domains D2 and D3.

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Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators Lancet Neurol....

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