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Recent Progress of Antisense Oligonucleotide Therapy for Superoxide Dismutase 1-mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen
Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disorder characterized by motor neuron degeneration, often leading to death within a few years. While there are limited treatments, genetic mutations in superoxide dismutase 1 (SOD1) have been identified in 2% of ALS cases. Tofersen, an antisense oligonucleotide (ASO), specifically targets SOD1 mRNA, reducing the production of toxic SOD1 protein and has shown promise in clinical trials, resulting in FDA approval in …
