From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis

Nucleic Acid Ther. 2023 Aug;33(4):234-237. doi: 10.1089/nat.2023.0027.
No abstract available

MeSH terms

  • Amyotrophic Lateral Sclerosis* / drug therapy
  • Amyotrophic Lateral Sclerosis* / genetics
  • Drug Approval
  • Endpoint Determination
  • Humans
  • Mutation
  • Oligonucleotides, Antisense* / therapeutic use
  • Superoxide Dismutase-1 / genetics
  • United States
  • United States Food and Drug Administration

Substances

  • Oligonucleotides, Antisense
  • SOD1 protein, human
  • Superoxide Dismutase-1
  • tofersen