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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Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2-4 (SPACE trial)

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