| Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia |
22 |
| Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis |
22 |
| A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors |
21 |
| The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab |
20 |
| Defining extended half-life rFVIIIA critical review of the evidence |
19 |
| Treatment of bleeding episodes in haemophilia A complicated by a factor VIII inhibitor in patients receiving Emicizumab. Interim guidance from UKHCDO Inhibitor Working Party and Executive Committee |
17 |
| Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project |
16 |
| Management of bleeding and invasive procedures in haemophilia A patients with inhibitor treated with emicizumab (Hemlibra (R)): Proposals from the French network on inherited bleeding disorders (MHEMO), the French Reference Centre on Haemophilia, in collaboration with the French Working Group on Perioperative Haemostasis (GIHP) |
15 |
| High proportion of patients with bleeding of unknown cause in persons with a mild-to-moderate bleeding tendency: Results from the Vienna Bleeding Biobank (VIBB) |
13 |
| Women with bleeding disorders |
13 |
| Recommendations on multidisciplinary management of elective surgery in people with haemophilia |
12 |
| Clinical use of recombinant factor VIII Fc and recombinant factor IX Fc in patients with haemophilia A and B |
12 |
| Recombinant factor VIII Fc fusion protein for immune tolerance induction in patients with severe haemophilia A with inhibitors-A retrospective analysis |
12 |
| Joint disease in haemophilia: Pathophysiology, pain and imaging |
12 |
| Once-weekly prophylaxis with glycoPEGylated recombinant factor VIII (N8-GP) in severe haemophilia A: Safety and efficacy results from pathfinder 2 (randomized phase III trial) |
11 |
| Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes |
11 |
| Use of telehealth in the delivery of comprehensive care for patients with haemophilia and other inherited bleeding disorders |
11 |
| Regional variation and cost implications of prescribed extended half-life factor concentrates among US Haemophilia Treatment Centres for patients with moderate and severe haemophilia |
11 |
| The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world |
10 |
| Acquired haemophilia in cancer: A systematic and critical literature review |
10 |
| Laboratory testing for factor VIII and IX inhibitors in haemophilia: A review |
10 |
| Genotypes, phenotypes and whole genome sequence: Approaches from the My Life Our Future haemophilia project |
10 |
| BAY 94- a PEGylated recombinant factor VIII, exhibits a prolonged half-life and higher area under the curve in patients with severe haemophilia A: Comprehensive pharmacokinetic assessment from clinical studies |
9 |
| Improved joint health in subjects with severe haemophilia A treated prophylactically with recombinant factor VIII Fc fusion protein |
9 |
| Emicizumab: Review of the literature and critical appraisal |
8 |
| Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study |
8 |
| Effects of pre-analytical heat treatment in factor VIII (FVIII) inhibitor assays on FVIII antibody levels |
8 |
| Mortality of patients with haemophilia in Brazil: First report |
8 |
| Recombinant factor VIII products and inhibitor development in previously untreated patients with severe haemophilia A: Combined analysis of three studies |
8 |
| First-year results of an expanded humanitarian aid programme for haemophilia in resource-constrained countries |
8 |
| Immune tolerance induction: What have we learned over time? |
7 |
| Vaccinations are not associated with inhibitor development in boys with severe haemophilia A |
7 |
| Evaluation of chromogenic factor IX assays by automated protocols |
7 |
| Real-world experience with use of Antihemophilic Factor (Recombinant), PEGylated for prophylaxis in severe haemophilia A |
7 |
| Real-life experience in switching to new extended half-life products at European haemophilia centres |
7 |
| Factor VIII activity of BAY 94-9027 is accurately measured with most commonly used assays: Results from an international laboratory study |
7 |
| The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study |
7 |
| Pattern of bleeding in a large prospective cohort of haemophilia A patients: A three-year follow-up of the AHEAD (Advate in HaEmophilia A outcome Database) study |
7 |
| How to discuss gene therapy for haemophilia? A patient and physician perspective |
7 |
| The diagnostic accuracy of bleeding assessment tools for the identification of patients with mild bleeding disorders: A systematic review |
7 |
| Individualized PK-based prophylaxis in severe haemophilia |
6 |
| Miracle of haemophilia drugs: Personal views about a few main players |
6 |
| Clinical evaluation of bleeds and response to haemostatic treatment in patients with acquired haemophilia: A global expert consensus statement |
6 |
| A field study evaluating the activity of N8-GP in spiked plasma samples at clinical haemostasis laboratories |
6 |
| Adherence to prophylactic haemophilic treatment in young patients transitioning to adult care: A qualitative review |
6 |
| Bleeding events and safety outcomes in persons with haemophilia A with inhibitors: A prospective, multi-centre, non-interventional study |
6 |
| The spectrum of bleeding in women and girls with haemophilia B |
6 |
| Impact of pain and functional impairment in US adults with haemophilia: Patient-reported outcomes and musculoskeletal evaluation in the pain, functional impairment and quality of life (P-FiQ) study |
6 |
| Spectrum and origin of mutations in sporadic cases of haemophilia A in China |
6 |
| The role of sclerostin/dickkopf-1 and receptor activator of nuclear factor kB ligand/osteoprotegerin signalling pathways in the development of osteoporosis in patients with haemophilia A and B: A cross-sectional study |
6 |
