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<span class="word">Longitudinal <span class="word allCaps">CSF <span class="word">and <span class="word">Serum <span class="word">Biomarker <span class="word">Dynamics <span class="word">in <span class="word">Tofersen <span class="word"><span class="changedDisabled">Treated <span class="word">SOD1-<span class="word allCaps">ALS: <span class="word"><span class="changedDisabled">A <span class="word"><span class="changedDisabled">Real-<span class="word"><span class="changedDisabled">World <span class="word"><span class="changedDisabled">Multicentre <span class="word"><span class="changedDisabled">Cohort <span class="word"><span class="changedDisabled">Study

2026-04-29

Abstract excerpt

Tofersen is a gene-targeted therapy for superoxide dismutase 1 (SOD1)-associated amyotrophic lateral sclerosis (ALS), but neurofilament light chain (NfL) may not fully capture the biological response to treatment. We performed a multicentre retrospective longitudinal study including 24 patients with SOD1-ALS treated with intrathecal tofersen at four Italian referral centres between 2022 and 2025. Cerebrospinal flu...

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Literature Corpus work: 1e6d3c0e-d544-54de-974b-908cf6d6008b
DOI: 10.20944/preprints202604.2098.v1

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<span class="word">Longitudinal <span class="word allCaps">CSF <span class="word">and <span class="word">Serum <span class="word">Biomarker <span class="word">Dynamics <span class="word">in <span class="word">Tofersen <span class="word"><span class="changedDisabled">Treated <span class="word">SOD1-<span class="word allCaps">ALS: <span class="word"><span class="changedDisabled">A <span class="word"><span class="changedDisabled">Real-<span class="word"><span class="changedDisabled">World <span cl…DOI 10.20944/preprints202604.2098.v1Read

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