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- Title
First FDA-Approved Gene Therapy for Hemophilia.
- Authors
Larkin, Howard
- Abstract
News From the Food and Drug Administration The FDA granted its first approval for a gene therapy for hemophilia in late November. Hemophilia B is caused by a gene defect that reduces or eliminates functional factor IX production, according to a Centers for Disease Control and Prevention fact page. Although the effectiveness of genetic therapy tends to decline over time, a modeling study suggested that more than 80% of patients with hemophilia B would not need prophylactic factor IX therapy for up to 25.5 years after 1 etranacogene dezaparvovec treatment.
- Subjects
UNITED States. Food &; Drug Administration; GENE therapy; BLOOD coagulation factor IX; HEMOPHILIA; BLOOD coagulation factors
- Publication
JAMA: Journal of the American Medical Association, 2023, Vol 329, Issue 1, p14
- ISSN
0098-7484
- Publication type
Article
- DOI
10.1001/jama.2022.23510