Neurofilament Blood Levels Likely to Predict Treatment Response in SMA

Neurofilament Blood Levels Likely to Predict Treatment Response in SMA

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Neurofilament Blood Levels Likely to Predict Treatment Response in SMA

In infants with spinal muscular atrophy (SMA) type 1, lower blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before starting treatment with Spinraza (nusinersen) are significantly associated with greater motor gains over time. These findings, based on data from the Phase 3 ENDEAR trial and the SHINE extension study, contrast with those previously reported for children with types 2 and 3 disease, in whom higher pNF-H blood levels at…

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