Center For Neurologic Study

Tofersen Shows Long-Term Efficacy in Rare Genetic ALS, Stabilizing Disease and Improving Function in Quarter of Patients

- Long-term data from a phase 3 trial shows tofersen, an FDA-approved drug for SOD1-ALS, delays symptom progression and death over 3-5 years of treatment. - Approximately 25% of patients experienced stabilization or improvement in strength and respiratory function, unprecedented outcomes in this devastating neurological disease. - The antisense oligonucleotide drug targets the SOD1 gene mutation affecting only 2% of ALS cases but provides proof-of-concept for treating other ALS forms. - Patients who started tofersen early showed a 48% reduction in risk of death within six months compared to those who received placebo initially.