SMA News Briefs

A new study evaluated the correlation and predictive power of several cerebrospinal fluid (CSF) biomarkers related to functional motor scores, disease activity, and clinical outcomes in patients with spinal muscular atrophy (SMA) before and during nusinersen (Spinraza®) treatment. Researchers analyzed the levels of phosphorylated heavy chain (pNf-H), light-chain neurofilaments (NfL), total tau protein (T-Tau), neurogranin,…

Pediatric and adult spinal muscular atrophy (SMA) type 3 patients showed improved Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM) scores after 12 months of treatment with nusinersen (Spinraza®). The “findings expand the available data on the effect of nusinersen on type 3 patients, so far mostly limited to adult patients,”…