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Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen
Tofersen is an FDA-approved antisense oligonucleotide therapy targeting SOD1 mutations in ALS. This treatment reduces toxic SOD1 protein levels, showing promise in slowing disease progression. Despite challenges with intrathecal administration, ongoing research explores improved delivery methods and earlier interventions for presymptomatic carriers.
