Neurodegenerative and neuroinflammatory changes in SOD1-ALS patients receiving tofersen

Neurodegenerative and neuroinflammatory changes in SOD1-ALS patients receiving tofersen

Abstract The initiation of tofersen, a new specific antisense oligonucleotide (ASO) for SOD1 pathology, marked a significant turning point for SOD1-ALS patients. While clinical trials and early access program studies reported a significant reduction in plasma and cerebrospinal fluid (CSF) neurofilam...

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Main Authors: Cecilia Simonini, Elisabetta Zucchi, Ilaria Martinelli, Giulia Gianferrari, Christian Lunetta, Gianni Sorarù, Francesca Trojsi, Roberta Pepe, Rachele Piras, Matteo Giacchino, Federico Banchelli, Jessica Mandrioli
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Scientific Reports
Subjects:
ALS
Tofersen
Neurofilaments
CHI3L1
SerpinA1
Online Access:https://doi.org/10.1038/s41598-025-94984-1
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https://doi.org/10.1038/s41598-025-94984-1

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