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Tofersen

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

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

Posted by By MedXY 07/08/2026
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.
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Long-Term Tofersen Therapy in SOD1-ALS: Early Initiation Preserves Function and Extends Survival
Posted inNeurology news

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

Posted by By MedXY 02/11/2026
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 follow-up period of up to 3.5 years.
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