Topic: pNfL

Objectives To determine whether plasma neurofilament light chain (pNfL) can be a reliable and minimally invasive biomarker for monitoring response in children with later-onset spinal muscular atrophy (SMA) undergoing nusinersen treatment.Methods Neurofilament light chain(NfL) levels were measured from plasma(pNfL) and cerebrospinal fluid (cNfL) of 40 children with later-onset SMA undergoing nusinersen treatment. A total of 480 CSF and plasma samples were taken during the first six doses of treatment from April 2021 to February 2023. Correlational analyses was used to determine the relationship