Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A
暂无分享,去创建一个
M. Laffan | K. Pasi | S. Rangarajan | G. Pierce | W. Lester | B. Madan | E. Symington | Benjamin Kim | Xinqun Yang | W. Y. Wong | N. Mitchell | Tara M Robinson | G. Pierce | T. Robinson | K. J. Pasi | Glenn F Pierce
[1] T. Buckner,et al. Evidence of a disability paradox in patient‐reported outcomes in haemophilia , 2021, Haemophilia : the official journal of the World Federation of Hemophilia.
[2] K. Pasi,et al. Activity of Transgene-Produced B-Domain Deleted Factor VIII in Human Plasma Following AAV5 Gene Therapy. , 2020, Blood.
[3] E. Santagostino,et al. WFH Guidelines for the Management of Hemophilia, 3rd edition , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.
[4] G. Pierce. Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.
[5] M. Laffan,et al. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. , 2020, The New England journal of medicine.
[6] L. Tang,et al. Impact of prophylaxis on health‐related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries , 2019, Research and practice in thrombosis and haemostasis.
[7] J. Oldenburg,et al. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting , 2019, Haemophilia : the official journal of the World Federation of Hemophilia.
[8] A. Iorio,et al. Impact of Adopting Population Pharmacokinetics for Tailoring Prophylaxis in Haemophilia A Patients: A Historically Controlled Observational Study , 2019, Thrombosis and Haemostasis.
[9] V. Blanchette,et al. The Jamaican Haemophilia Registry: Describing the burden of disease , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.
[10] D. Perry,et al. AAV5–Factor VIII Gene Transfer in Severe Hemophilia A , 2017, The New England journal of medicine.
[11] C. Peterfy,et al. Effect of late prophylaxis in hemophilia on joint status: a randomized trial , 2017, Journal of thrombosis and haemostasis : JTH.
[12] P. Camp,et al. Patient powered prophylaxis: A 12‐month study of individualized prophylaxis in adults with severe haemophilia A , 2017, Haemophilia : the official journal of the World Federation of Hemophilia.
[13] C. Terwee,et al. Health‐related quality of life questionnaires in individuals with haemophilia: a systematic review of their measurement properties , 2017, Haemophilia : the official journal of the World Federation of Hemophilia.
[14] R. Kotin,et al. Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines. , 2017, Human gene therapy.
[15] Carter,et al. Levels of Human FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice Following Gene Therapy with BMN 270 , 2017 .
[16] B. Riske,et al. Men with severe hemophilia in the United States: birth cohort analysis of a large national database. , 2016, Blood.
[17] V. Blanchette,et al. Generic and disease-specific quality of life among youth and young men with Hemophilia in Canada , 2016, BMC Hematology.
[18] K. Wyrwich,et al. Interpreting important health‐related quality of life change using the Haem‐A‐QoL , 2015, Haemophilia : the official journal of the World Federation of Hemophilia.
[19] Michael Recht,et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. , 2014, The New England journal of medicine.
[20] A. Iorio,et al. Assessment of the impact of treatment on quality of life of patients with haemophilia A at different ages: insights from two clinical trials on turoctocog alfa , 2014, Haemophilia : the official journal of the World Federation of Hemophilia.
[21] J. Mahlangu,et al. Guidelines for the management of hemophilia , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.
[22] Pratima Chowdary,et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. , 2011, The New England journal of medicine.
[23] F. Rosendaal,et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels , 2011, Haemophilia.
[24] M. Bullinger,et al. Cross‐cultural development and psychometric evaluation of a patient‐reported health‐related quality of life questionnaire for adults with haemophilia , 2008, Haemophilia : the official journal of the World Federation of Hemophilia.