How to Best Measure Disease Progression in Adult Spinal Muscular Atrophy Patients: A Clinical and Neurophysiological Study
da Graça FF, Iwabe C, Nucci A, de Rezende TJR, França MC Jr.
Muscle Nerve 2026 Feb 2. doi: 10.1002/mus.70169
This study aimed to identify the most sensitive clinical, patient-reported, and neurophysiological measures to detect short-term disease progression in untreated adult SMA patients. Amongst every clinician-reported outcomes (MFM, HFMSE, RULM) and patient-reported outcomes (MFIS, CMAP, MUNIX), the MFM was the most sensitive to short-term progression and most closely aligned with neurophysiological markers, supporting its use as primary outcome in clinical trials involving adult SMA patients.
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