Molecular Biomarkers for Spinal Muscular Atrophy: A Systematic Review

Conclusion There is no sufficient evidence to support the clinical utility of SMN-related biomarkers to predict disease severity in SMA. pNF-H appears to be a promising biomarker of disease activity and treatment effect in SMA. Further studies should include longitudinal assessments of patients with SMA across functional groups and comparisons with age-matched healthy controls to evaluate the stability of putative biomarkers over time and in response to SMA therapeutics. PROSPERO registration: CRD42019139050.
Source: Neurology Clinical Practice - Category: Neurology Authors: Tags: All Clinical Neurology, Prognosis Review Source Type: research