Last Posted: Sep 16, 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
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
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