Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy

Hum Gene Ther. 2024 Apr;35(7-8):215-218. doi: 10.1089/hum.2024.29268.bfs.
No abstract available

MeSH terms

  • Cerebroside-Sulfatase / genetics
  • Genetic Therapy
  • Humans
  • Leukodystrophy, Metachromatic* / genetics
  • Leukodystrophy, Metachromatic* / therapy

Substances

  • Cerebroside-Sulfatase