Neurofilaments in cerebrospinal fluid and/or serum as a possible biomarker of pediatric spinal muscular atrophy. (PubMed, J Neuromuscul Dis) - Oct 18, 2026 - "Although therapies such as nusinersen have altered disease trajectories, objective biomarkers for treatment monitoring remain lacking...Longitudinal changes are more informative than single measurements. Further validation in larger cohorts and using more sensitive methods is warranted."
Biomarker • Journal • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases • NEFH
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Barbora Lauerova; Damjan Osredkar; Eva Vrs?aj; Jana Haberlová; Jitka Hanzalova; Marie Rohlenová; Markéta Kumhera; Marketa Pavlikova; Petra Holotova; Tanja Loboda; Vanesa Jurášová
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