Responsiveness of the Motor Function Measure in Patients With Spinal Muscular Atrophy.

Vuillerot  C, Payan C, Iwaz J, Ecochard R, Bérard C and the MFM Spinal Muscular Atrophy Study Group.
Arch Phys Med Rehabil. 2013. 94(8) : 1555-61.
Assessment in 112 patients with Spinal Muscular Atrophy (SMA) aged 5.7 to 59 years of the ability of the MFM to detect changes in the progression of SMA. In patients with SMA type 2 and type 3, there was a moderate inverse relationship between age and the MFM total score. Patients with less than 6 months’ follow-up showed little score changes. Patients with a longer follow-up showed a slow deterioration (-0.9 points/y for type 2 and -0.6 points/y for type 3). Substantial responsiveness was obtained with the MFM D2 subscore (proximal and axial motricity) in patients with SMA type 2, and with the MFM D1 subscore (standing and transfers) in patients with SMA type 3.

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Keywords: Clinical trial, Mobility limitation, Muscle weakness, Spinal Muscular Atrophy, Neuromuscular diseases, Rehabilitation