Article

Targeted proteomics upon Tofersen identifies candidate response markers for SOD1-linked amyotrophic lateral sclerosis

2024-04-24

Abstract excerpt

Tofersen is the first effective and approved therapy for familial ALS caused by pathogenic variants in the SOD1 gene. Following treatment with tofersen, neurofilaments in patients CSF and serum display a faster response than clinical parameters, underlining their importance as a biomarker for treatment response in clinical trials. This evidence led us to hypothesize that this novel treatment might represent an opp...

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Literature Corpus work: adad78ac-d2b4-5112-bb1c-846b675bc19c
DOI: 10.1101/2024.04.22.24306165

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