Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy

Trundell D, Le Scouiller S, Le Goff L, Gorni K, Vuillerot C.
PLoS One. 2020. 18;15(9):e0238786.
This study sought to investigate the reliability, validity, and ability to detect change of MFM32 in subjects with Type 2 and non-ambulant Type 3 Spinal Muscular Atrophy (SMA). Data from 110 sujbects originally included in a Phase 2 study assessing the efficacy and safety of olesoxime were analyzed.
Results support the use of the MFM32 in longitudinal research in individuals with Type 2 and non-ambulant Type 3 SMA.

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