Last Posted: Jun 24, 2021
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
- Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.
Konkle Barbara et al. Journal of thrombosis and haemostasis : JTH 2020 Nov 18(11) 3074-3077
- Current Clinical Applications of in vivo Gene Therapy with AAVs.
Mendell Jerry R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Dec
- Assessing the current knowledge, attitude and behaviour of adolescents and young adults living with haemophilia.
Mohan Richa et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Dec
- Addressing patient education priorities in the era of gene therapy for haemophilia: Towards evidence-informed shared decision-making.
Woollard Laurence et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Nov
- Reimbursing the value of gene therapy care in an era of uncertainty.
Noone Declan et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Nov
- Evaluation of anti-factor VIII antibody levels in patients with haemophilia A receiving immune tolerance induction therapy or bypassing agents.
Boylan Brian et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Nov
- Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.
van Overbeeke Eline et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Nov
- Insights from the Hereditary Thrombotic Thrombocytopenic Purpura Registry: Discussion of Key Findings Based on Individual Cases from Switzerland.
Kremer Hovinga Johanna A et al. Hamostaseologie 2020 Nov 40(S 01) S5-S14
- Novel F8 and F9 gene variants from the PedNet Hemophilia Registry classified according to ACMG/AMP guidelines.
Andersson Nadine G et al. Human mutation 2020 Sep
- Treatment preferences in people with haemophilia A or caregivers of people with haemophilia A: A discrete choice experiment.
Fifer Simon et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Aug 26 Suppl 530-40
- Evaluation of CDC's Hemophilia Surveillance Program - Universal Data Collection (1998-2011) and Community Counts (2011-2019), United States.
Schieve Laura A et al. Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002) 2020 Sep (5) 1-18
- BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
Konkle Barbara A et al. The New England journal of medicine 2020 Sep (11) 1018-1027
- Prevalence of FVIII Inhibitors Among Children with Hemophilia A: Experience at the Jordanian Royal Medical Services.
Oudat Raida et al. Medical archives (Sarajevo, Bosnia and Herzegovina) 2020 Jun 74(3) 187-190
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