Open Access
Issue |
Med Sci (Paris)
Volume 39, Number 2, Février 2023
|
|
---|---|---|
Page(s) | 187 - 190 | |
Section | Forum | |
DOI | https://doi.org/10.1051/medsci/2023003 | |
Published online | 17 February 2023 |
- Franchini M, Mannucci PM. Past, present and future of hemophilia: a narrative review. Orphanet J Rare Dis 2012 ; 7 : 24. [CrossRef] [PubMed] [Google Scholar]
- Jordan B. Hémophilie : la thérapie génique, enfin…. Med Sci (Paris) 2018 ; 34 : 267–270. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
- Batty P, Lillicrap D. Hemophilia Gene Therapy: Approaching the First Licensed Product. Hemasphere 2021; 5 : e540. [CrossRef] [PubMed] [Google Scholar]
- Pipe S, Giermasz A, Castaman G, et al. One year data from a phase 2b trial of AMT-061 (AAV5-Padua hFIX variant), an enhanced vector for gene transfer in adults with severe or moderate-severe hemophilia B. Blood 2019 ; 134 : 3348. [CrossRef] [Google Scholar]
- von Drygalski A, Gomez E, Giermasz A, et al. Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy. Blood Adv 2022; bloodadvances.2022008886. [PubMed] [Google Scholar]
- https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b. [Google Scholar]
- Naddaf M. Researchers welcome $3.5-million haemophilia gene therapy — but questions remain. Nature 2022; 612 : 388–9. [CrossRef] [PubMed] [Google Scholar]
- Jordan B. Immunothérapie « CAR-T » : une autorisation qui fait date. Med Sci (Paris) 2017 ; 33 : 1003–1006. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
- Li N, Sawyer EK, Maruszczyk K, et al. Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model. J Med Econ 2021; 24 : 363–72. [CrossRef] [PubMed] [Google Scholar]
- https://icer.org/news-insights/press-releases/icer-publishes-evidence-report-on-gene-therapies-for-hemophilia-a-and-b/. [Google Scholar]
- https://assurance-maladie.ameli.fr/etudes-et-donnees/2006-cout-ald-2004. [Google Scholar]
- Jordan B. Une approche pragmatique pour gérer les traitements onéreux. Med Sci (Paris) 2017 ; 33 : 1121–1123. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
Current usage metrics show cumulative count of Article Views (full-text article views including HTML views, PDF and ePub downloads, according to the available data) and Abstracts Views on Vision4Press platform.
Data correspond to usage on the plateform after 2015. The current usage metrics is available 48-96 hours after online publication and is updated daily on week days.
Initial download of the metrics may take a while.