ArticleActa neurologica Belgica2025
Cerebrospinal fluid biomarkers of efficacy in patients affected by spinal muscular atrophy type 1 treated with nusinersen.
Article in Acta neurologica Belgica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
3 citing papers in PubMed.
- The role of neurofilament light chain and other fluid biomarkers in cerebellar ataxias.Brain communications · 2026Review
- Biomarkers in spinal muscular atrophy.Frontiers in neurology · 2025Review
- Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study.Frontiers in neurologyArticle
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe advent of new therapies, such as the antisense oligonucleotide nusinersen, has significantly improved the natural course of spinal muscular atrophy (SMA). Tau proteins and neurofilaments are well known markers of neuro-axonal damages. The neurofilament light protein (NfL) has been proposed as a possible biomarker in SMA. This study aimed to investigate the role of total-tau (t-tau), phosphorylated tau at 181 sites (p-tau 181), NfL, and phosphorylated neurofilament heavy chain (pNfH) proteins as potential cerebrospinal fluid (CSF) biomarkers of disease severity and/or nusinersen treatment response in 14 SMA type 1 patients with a wide age range (2-156 months). METHODS AND
resultsMotor functions were assessed using the "Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders" (CHOP-INTEND) scale at baseline, six months and ten months after treatment. Eight out of 14 patients showed motor improvement. At baseline CSF t-tau and p-tau181 concentration showed a significant negative correlation with age (p = 0.0002 and p = 0.0054 respectively) and a positive correlation with the CHOP-INTEND score (p = 0.0075 and p = 0.0342, respectively). After treatment the tau biomarkers did not show any change, whereas NfL and pNfH concentration significantly decreased (p = 0.0001). The NfL concentration decline correlated to age at baseline (p < 0.05) and to the improvement of the CHOP-INTEND motor score, in the subgroup of patients with a functional improvement above 3 points (p < 0.05).
conclusionsCSF neurofilaments and particularly NfL may bepromising biomarkers for monitoring treatment response to nusinersen, both in younger and older patients with severe SMA.
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40317413What Socratic holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.