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This single-center observational study evaluated 8 adults with genetically confirmed SOD1 p.Leu145Phe ALS treated with intrathecal tofersen. All had lower-limb onset and a predominantly lower motor neuron phenotype. Median age at symptom onset was 60 years and treatment delay was 48 months. The median on-treatment ALSFRS-R decline was −0.28 points/month (range +0.04 to −0.57); 2 patients remained stable, while others showed gradual decline. No serious treatment-related adverse events occurred.
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This single-center observational study evaluated 8 adults with genetically confirmed SOD1 p.Leu145Phe ALS treated with intrathecal tofersen. All had lower-limb onset and a predominantly lower motor neuron phenotype. Median age at symptom onset was 60 years and treatment delay was 48 months. The median on-treatment ALSFRS-R decline was −0.28 points/month (range +0.04 to −0.57); 2 patients remained stable, while others showed gradual decline. No serious treatment-related adverse events occurred.
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