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This systematic review and meta-analysis evaluates the safety and efficacy of tofersen, an antisense oligonucleotide approved for treating SOD1-related amyotrophic lateral sclerosis (ALS). The analysis included 12 studies with 195 patients, revealing that tofersen significantly reduces SOD1 levels in cerebrospinal fluid and slows disease progression, as indicated by improved ALS Functional Rating Scale scores. Adverse events were consistent with ALS progression, suggesting that tofersen is a promising targe

Author

magec72802

Language

EN