Last Posted: Nov 30, 2021
- Gene therapy for hemophilia A
K O'Leary, Nature Medicine, November 30, 2021
- Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
LA George et al, NEJM, November 18, 2021
- Real Stories from People Living with Hemophilia – Carrie
CDC, November 2021
- Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations.
Garrison Louis P et al. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research 2021 24(11) 1628-1633
- Why is AAV FVIII gene therapy not approved by the US Food and Drug Administration yet?
Arruda Valder R et al. Blood advances 2021 5(20) 4313
- Emerging drugs for hemophilia A: insights into phase II and III clinical trials.
Kizilocak Hande et al. Expert opinion on emerging drugs 2021
- What is a ‘serious’ genetic condition? The perceptions of people living with genetic conditions
FK Boardman et al, EJHG, September 27, 2021
- [Current developments in hemostatic treatment for patients with hemophilia].
Nogami Keiji et al. [Rinsho ketsueki] The Japanese journal of clinical hematology 2021 62(8) 1213-1221
- A qualitative study on the experiences of haemophilia carriers before, during and after pregnancy.
Punt Marieke C et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
- Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Hemophilia B Without Inhibitors in China.
Liu Guoqing et al. Clinical therapeutics 2021
- Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A.
Pasi K John et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
- Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment.
Witkop Michelle et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
- Gene therapy for hemophilia: Current status and laboratory consequences.
Batty Paul et al. International journal of laboratory hematology 2021 43 Suppl 1117-123
E Berntorp et al, Nature Dis Primers., June 24, 2021
- Prediction of hemophilia A severity using a small-input machine-learning framework.
Lopes Tiago J S et al. NPJ systems biology and applications 2021 7(1) 22
- Patterns of joint damage in severe haemophilia A treated with prophylaxis.
Goren Rachel et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
- Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.
Hart Daniel P et al. Orphanet journal of rare diseases 2021 16(1) 189
- Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
Garrison Louis P et al. Journal of managed care & specialty pharmacy 2021 27(5) 674-681
- Data from CDC’s hemophilia surveillance programs can help prevent or reduce hemophilia-related health problems
CDC, March 2021
- Health care resource utilization and costs among adult patients with hemophilia A on factor VIII prophylaxis: an administrative claims analysis.
Croteau Stacy E et al. Journal of managed care & specialty pharmacy 2021 27(3) 316-326
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