P.273Serum neurofilament light chain in type 1 spinal muscular atrophy: 30 months data from first part of a branaplam phase II study

Results from patients with neurodegenerative diseases have shown that neurofilament light chain (NfL) is a sensitive, blood-based marker that can identify patients, aid in prediction of long-term outcomes, and be used to assess effects of treatment. At present, little is known about NfL levels in the blood of patients with spinal muscular atrophy (SMA). The present study assesses the relevance of serum NfL as a potential biomarker for SMA Type I...