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WCN 2021 | Novel outcome measures for ALS and SMA

Marianne de Visser, MD, Academic Medical Centre (AMC), University of Amsterdam, Amsterdam, The Netherlands, talks on the development of novel outcome measures for amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Traditional outcome measures used in ALS trials are limited by large sample size requirements. A new promising biomarker, neurofilament-light chain (NF-L), has been found in elevated levels in the cerebrospinal fluid (CSF) of patients with early-stage ALS and could dramatically reduce the sample sizes needed for clinical trials. Similarly, plasma phosphorylated neurofilament heavy chain (pNF‐H) could be used as a biomarker in spinal muscular atrophy type 1 (SMA1). This interview took place during the XXV World Congress of Neurology.


Consultancy with Novartis Therapeutics and Dynacure.