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Neurology

Decoding Tofersen’s CNS Penetrance and Efficacy in SOD1-ALS Through Autopsy Tissue Analysis

This study unveils tofersen’s distribution and potent SOD1 reduction within the CNS of SOD1-ALS patients, utilizing autopsy tissues from clinical trial participants, confirming anticipated pharmacokinetic and pharmacodynamic effects.

Jul 8, 2026
Neurology

Long-Term Tofersen Therapy in SOD1-ALS: Early Initiation Preserves Function and Extends Survival

The integrated analysis of the VALOR trial and its open-label extension reveals that early initiation of tofersen significantly slows functional decline, preserves respiratory capacity, and improves survival in patients with SOD1-ALS over a

Feb 11, 2026

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