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- Publisher
- Springer Journals
- Copyright
- Copyright © Springer Nature America, Inc. 2022
- ISSN
- 1078-8956
- eISSN
- 1546-170X
- DOI
- 10.1038/s41591-022-01934-9
- Publisher site
- See Article on Publisher Site
Abstract
Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several primary immunodeficiencies, and yet companies in this space are withdrawing from the EU market. Technological and regulatory innovations and a change to cost–benefit models are needed so that rare disease patients can receive these life-saving medicines.
Journal
Nature Medicine – Springer Journals
Published: Oct 1, 2022