Keywords: Muscle, Pediatric, neuromuscular disease
Motivation: A biomarker to assess the efficacy of Nusinersen is needed as it enhances motor function and prolongs survival in SMA patients.
Goal(s): We investigated whether qMRI could monitor the efficacy of Nusinersen in the treatment of SMA.
Approach: Patients underwent measurements of thigh qMRI (mDixon-Quant, T2 mapping and DTI) and Hammersmith Functional Motor Scale Expanded (HFMSE) before and after nusinersen treatment, X-ray exams to obtain Cobb angle were performed at baseline.
Results: After treatment, the mean thigh FF and FA decreased over time, and the HFMSE increased, while T2 values and ADC remain stable.
Impact: qMRI can evaluate the treatment outcomes of SMA patients. The younger patients with less scoliosis more likely to have minimumclinically significant difference at 6 months after treatment.
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