Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

Evidence of a disability paradox in patient‐reported outcomes in haemophilia

(2021) Jamie O’Hara et al.   HAEMOPHILIA

Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns

(2020) Glenn F. Pierce   HAEMOPHILIA

WFH Guidelines for the Management of Hemophilia, 3rd edition

(2020) Alok Srivastava et al.   HAEMOPHILIA

Activity of Transgene-Produced B-Domain Deleted Factor VIII in Human Plasma Following AAV5 Gene Therapy

(2020) Steffen Rosen et al.   BLOOD

Impact of Adopting Population Pharmacokinetics for Tailoring Prophylaxis in Haemophilia A Patients: A Historically Controlled Observational Study

(2019) Felix Kallenbach et al.   THROMBOSIS AND HAEMOSTASIS

Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting

(2019) Rebecca Kruse‐Jarres et al.   HAEMOPHILIA

The Jamaican Haemophilia Registry: Describing the burden of disease

(2018) G. Wharfe et al.   HAEMOPHILIA

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

(2018) Stuart Bunting et al.   MOLECULAR THERAPY

Health-related quality of life questionnaires in individuals with haemophilia: a systematic review of their measurement properties

(2017) P.F. Limperg et al.   HAEMOPHILIA

Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A

(2017) Haowei Linda Sun et al.   HAEMOPHILIA

Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines

(2017) Robert M. Kotin et al.   HUMAN GENE THERAPY

Effect of late prophylaxis in hemophilia on joint status: a randomized trial

(2017) M. J. Manco-Johnson et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

(2016) M. A. Mazepa et al.   BLOOD

Interpreting important health-related quality of life change using the Haem-A-QoL

(2015) K. W. Wyrwich et al.   HAEMOPHILIA

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) E. Santagostino et al.   HAEMOPHILIA

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Guidelines for the management of hemophilia

(2012) A. Srivastava et al.   HAEMOPHILIA

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels

(2010) I. E. M. DEN UIJL et al.   HAEMOPHILIA

Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia

(2008) A. RENTZ et al.   HAEMOPHILIA