Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis
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Importance: Approximately 2% of amyotrophic lateral sclerosis (ALS) cases are attributable to a pathogenic variant in the superoxide dismutase 1 (SOD1) gene. Tofersen, an intrathecal antisense oligonucleotide designed to reduce S... ...
