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.
Articles in scientific journal
Motor Function Measure: portuguese version and relability analysis.
Iwabe C., Miranda-Pfeilsticker BH., Nucci A. Rev Bras Fisioter 2008. 24 : 15-19 The aim of the study was to translate in Portuguese the MFM and to identify its intra and inter-examiner reliability in Brazilian patients.Keywords: neuromuscular disorders, motor...