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.
Author
Alex Philippidis
1
Affiliation
1
Mary Ann Liebert, Inc., Publishers, New Rochelle, New York, USA.
PMID:
38626339
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