Are You a Healthcare Professional?
This European website, initiated and developed by CSL Behring, has two separate sections with the aim to provide information on haemophilia for an international audience, either to European healthcare professionals or to the general public.*
Yes, I am a healthcare professional*
No, I am not a healthcare professional
- Miesbach W, Chowdary P, Coppens M, et al. Delivery of AAV-based gene therapy through haemophilia centers-a need for re-evaluation of infrastructure and comprehensive care: a joint publication of EAHAD and EHC. Haemophilia. 2021;27:967-973.
- Krumb E, Lambert C, Hermans C. Patient selection for hemophilia gene therapy: real-life data from a single center. Res Pract Thromb Haemost. 2021;5(3):390-394.
- Nathwani AC, Tuddenham EGD, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. New Engl J Med. 2011;365(25):2357-2365.
- Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII gene transfer in severe hemophilia A. New Engl J Med. 2017;377(26):2519-2530.
- European Medicines Agency (EMA). Roctavian. https://www.ema.europa.eu/en/medicines/human/EPAR/roctavian-0. Accessed January 28, 2023.
- European Medicines Agency (EMA). Hemgenix. https://www.ema.europa.eu/en/medicines/human/EPAR/hemgenix. Accessed July 14, 2023.
- Food and Drug Administration. Approved Cellular and Gene Therapy Products. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products. Accessed September 5, 2023.
- EAHAD and ISTH. ISTH and EAHAD educational partnership on gene therapy. https://eahad.org/isth-and-eahad-educational-partnership-on-gene-therapy/. Accessed October 20, 2021.
- Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.