Tofersen
New Drug Approvals
MAY 17, 2025
3] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1 , an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. 3] Tofersen was approved for medical use in the United States in April 2023, [3] [6] and in the European Union in May 2024. [4] 3 November 2006.
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