Abstract

Tofersen (Qalsody – Biogen), an intrathecally administered antisense oligonucleotide, has received accelerated approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

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Topics from this Paper

  • Amyotrophic Lateral Sclerosis
  • Superoxide Dismutase 1
  • Antisense Oligonucleotide

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