scholarly article | Q13442814 |
P356 | DOI | 10.1016/S0140-6736(15)01123-X |
P698 | PubMed publication ID | 26897598 |
P50 | author | Flora Peyvandi | Q39183632 |
P2093 | author name string | Guy Young | |
Isabella Garagiola | |||
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Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. | Q45880382 | ||
Analysis of mRNA in hemophilia A patients with undetectable mutations reveals normal splicing in the factor VIII gene. | Q45880854 | ||
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Recurrent inversion breaking intron 1 of the factor VIII gene is a frequent cause of severe hemophilia A. | Q45882168 | ||
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model | Q45882197 | ||
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Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China | Q45883686 | ||
Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. | Q45884927 | ||
Identification of de novo deletion in the factor VIII gene by MLPA technique in two girls with isolated factor VIII deficiency | Q45885124 | ||
Concomitant infusion of low doses of rFVIIa and FEIBA in haemophilia patients with inhibitors | Q45885613 | ||
Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. | Q45886344 | ||
Identification of factor VIII gene mutations in 101 patients with haemophilia A: mutation analysis by inversion screening and multiplex PCR and CSGE and molecular modelling of 10 novel missense substitutions | Q45886376 | ||
Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. | Q45886921 | ||
Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. | Q45889455 | ||
Rituximab for treatment of inhibitors in haemophilia A. A Phase II study. | Q49866575 | ||
Aptamers as therapeutics. | Q51813594 | ||
Haemophilia A mutations in patients with non-severe phenotype associated with a discrepancy between one-stage and chromogenic factor VIII activity assays. | Q53071200 | ||
Inhibitor development in non-severe haemophilia across Europe. | Q53378171 | ||
Presence of fetal DNA in maternal plasma and serum | Q57075132 | ||
Inhibitor development in haemophilia according to concentrate | Q59468965 | ||
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Characterisation of 96 mutations in 128 unrelated severe haemophilia A patients from France. Description of 62 novel mutations | Q83120292 | ||
Fetal loss rate after chorionic villus sampling and amniocentesis: an 11-year national registry study | Q84018877 | ||
Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial | Q34737483 | ||
Long-term safety and efficacy of factor IX gene therapy in hemophilia B. | Q34783922 | ||
Consensus perspectives on prophylactic therapy for haemophilia: summary statement | Q35112424 | ||
Standardization of FVIII & FIX assays | Q35163622 | ||
Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A. | Q35181684 | ||
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
A cross-sectional study of bleeding phenotype in haemophilia A carriers | Q35812405 | ||
Hemophilia B: molecular pathogenesis and mutation analysis. | Q35832802 | ||
Hemophilic arthropathy | Q35912735 | ||
Monitoring haemophilia severity and treatment: new or old laboratory tests? | Q35915695 | ||
Inhibitors in congenital coagulation disorders | Q35937076 | ||
Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. | Q36250920 | ||
Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients | Q36380181 | ||
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant | Q36797248 | ||
Sequential therapy with activated prothrombin complex concentrates and recombinant FVIIa in patients with severe haemophilia and inhibitors: update of our previous experience | Q36819965 | ||
How to interpret and pursue an abnormal prothrombin time, activated partial thromboplastin time, and bleeding time in adults | Q36867591 | ||
Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up | Q37175598 | ||
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. | Q37489013 | ||
Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide | Q37634856 | ||
Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review | Q37719683 | ||
Blood-induced joint disease: the pathophysiology of hemophilic arthropathy | Q37768520 | ||
Discrepancy between one-stage and chromogenic factor VIII activity assay results can lead to misdiagnosis of haemophilia A phenotype. | Q37807879 | ||
Guideline on the management of haemophilia in the fetus and neonate | Q37873540 | ||
Parallel use of by-passing agents in haemophilia with inhibitors: a critical review | Q37927122 | ||
The diagnosis and management of congenital hemophilia | Q38046535 | ||
More than a decade of international experience with a pdFVIII/VWF concentrate in immune tolerance | Q38070812 | ||
Factor VIII inhibitors in hemophilia A: rationale and latest evidence | Q38101041 | ||
FEIBA versus NovoSeven in hemophilia patients with inhibitors | Q38135321 | ||
Hemophilia a and hemophilia B: different types of diseases? | Q38135323 | ||
Arthropathy in patients with moderate hemophilia a: a systematic review of the literature | Q38136140 | ||
Inhibitor eradication with rituximab in haemophilia: where do we stand? | Q38195908 | ||
Current status of haemophilia gene therapy | Q38206852 | ||
Innovative approach for improved rFVIII concentrate | Q38207134 | ||
Prophylaxis with FEIBA in paediatric patients with haemophilia A and inhibitors | Q38325328 | ||
1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases | Q39433082 | ||
Personalized prophylaxis | Q39596251 | ||
Inhibitor development and mortality in non-severe hemophilia A. | Q41014569 | ||
Impact of inhibitors on hemophilia A mortality in the United States. | Q41524981 | ||
Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. | Q41975229 | ||
The Fc gamma receptor IIa R131H polymorphism is associated with inhibitor development in severe hemophilia A. | Q42211106 | ||
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study | Q42598903 | ||
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. | Q42709633 | ||
Factor VIII products and inhibitor development in severe hemophilia A. | Q43770114 | ||
"Founder" effect in different families with haemophilia B mutation | Q43815426 | ||
Changing patterns of bleeding in patients with severe haemophilia A. | Q44584056 | ||
F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity | Q44646272 | ||
Physical functioning in boys with hemophilia in the U.S. | Q44715249 | ||
Inhibitors occur more frequently in African-American and Latino haemophiliacs | Q45855283 | ||
Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A. | Q45855813 | ||
Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. | Q45855994 | ||
Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile | Q45856559 | ||
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. | Q45858690 | ||
Clinical use of a new glycine-precipitated antihemophilic fraction | Q45859062 | ||
Production of High-Potency Concentrates of Antihemophilic Globulin in a Closed-Bag System | Q45859073 | ||
Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate. | Q45859168 | ||
Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. | Q45861960 | ||
Spectrum of molecular defects and mutation detection rate in patients with mild and moderate hemophilia A. | Q45862416 | ||
The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. A double-blind clinical trial | Q45864000 | ||
Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A. | Q45865637 | ||
Similar bleeding phenotype in young children with haemophilia A or B: a cohort study | Q45865672 | ||
Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. | Q45865894 | ||
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). | Q45866554 | ||
Trends in bleeding patterns during prophylaxis for severe haemophilia: observations from a series of prospective clinical trials | Q45866876 | ||
Comparing bleed frequency and factor concentrate use between haemophilia A and B patients | Q45867613 | ||
Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. | Q45868849 | ||
AIDS, hepatitis and hemophilia in the 1980s: memoirs from an insider | Q45869176 | ||
Non-invasive tool for foetal sex determination in early gestational age. | Q45869375 | ||
Phenotypic heterogeneity in severe hemophilia | Q45869550 | ||
Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors | Q45870481 | ||
A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group | Q45870861 | ||
Prenatal diagnosis and preimplantation genetic diagnosis: novel technologies and state of the art of PGD in different regions of the world | Q45871116 | ||
European study on orthopaedic status of haemophilia patients with inhibitors | Q45871281 | ||
Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial | Q45872638 | ||
Single 270 microg kg(-1)-dose rFVIIa vs. standard 90 microg kg(-1)-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison | Q45873703 | ||
Evaluation of DHPLC in the analysis of hemophilia A. | Q45874440 | ||
Identification of 217 unreported mutations in the F8 gene in a group of 1,410 unselected Italian patients with hemophilia A. | Q45874679 | ||
Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors | Q45874687 | ||
The principal results of the International Immune Tolerance Study: a randomized dose comparison | Q45875180 | ||
Neonatal bleeding in haemophilia: a European cohort study | Q45875593 | ||
The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. | Q45876258 | ||
P433 | issue | 10040 | |
P407 | language of work or name | English | Q1860 |
P921 | main subject | hemophilia | Q134003 |
P304 | page(s) | 187-197 | |
P577 | publication date | 2016-02-18 | |
P1433 | published in | The Lancet | Q939416 |
P1476 | title | The past and future of haemophilia: diagnosis, treatments, and its complications | |
P478 | volume | 388 |
Q92044581 | "The times they are a-changin", ... or maybe not? |
Q36356147 | Acquired hemophilia A: a review of recent data and new therapeutic options |
Q38945123 | Advances in the treatment of bleeding disorders. |
Q45873084 | Analyses of the FranceCoag cohort support immunogenicity differences among one plasma-derived and two recombinant factor VIII brands in boys with severe hemophilia A. |
Q57289034 | Anti-tissue factor pathway inhibitor (TFPI) therapy: a novel approach to the treatment of haemophilia |
Q97519097 | B Cell Depletion Eliminates FVIII Memory B Cells and Enhances AAV8-coF8 Immune Tolerance Induction When Combined With Rapamycin |
Q38941572 | BAY 81-8973 (Octocog Alfa; Kovaltry®): A Review in Haemophilia A. |
Q90516253 | Blood coagulation dissected |
Q88732008 | Blood-induced bone loss in murine hemophilic arthropathy is prevented by blocking the iRhom2/ADAM17/TNF-α pathway |
Q90674195 | Clinical utility and impact of the use of the chromogenic vs one-stage factor activity assays in haemophilia A and B |
Q39042380 | Coagulation Factor IX (Recombinant), Albumin Fusion Protein (Albutrepenonacog Alfa; Idelvion®): A Review of Its Use in Haemophilia B. |
Q92433809 | Comparison of 90-Day Complication Rates and Cost Between Single and Multiple Joint Procedures for End-Stage Arthropathy in Patients with Hemophilia |
Q104289829 | Current Clinical Applications of in vivo Gene Therapy with AAVs |
Q91788836 | Developing a new scoring scheme for the Hemophilia Joint Health Score 2.1. |
Q28075679 | Different impact of factor VIII products on inhibitor development? |
Q64046774 | Drug discovery and development for rare genetic disorders |
Q38720560 | Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa). |
Q38917306 | Efmoroctocog Alfa: A Review in Haemophilia A. |
Q38711095 | Eftrenonacog Alfa: A Review in Haemophilia B. |
Q38822601 | Elucidation of Factor VIII Activity Pharmacokinetics: A Pooled Population Analysis in Patients With Hemophilia A Treated With Moroctocog Alfa |
Q93073557 | Emicizumab for the treatment of haemophilia A: a narrative review |
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Q36290952 | Genome editing for inborn errors of metabolism: advancing towards the clinic. |
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Q55346403 | Inhibition of Tissue Factor Pathway Inhibitor (TFPI) as a Treatment for Haemophilia: Rationale with Focus on Concizumab. |
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Q45874959 | Modern Treatments of Haemophilia: Review of Cost-Effectiveness Analyses and Future Directions |
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Q39027887 | New therapies for hemophilia |
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Q45874270 | Nonacog Beta Pegol: A Review in Haemophilia B. |
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Q50610450 | [Retrospectively analysis of the difference of bleeding frequency and hemophilic arthropathy between hemophilia A and hemophilia B patients]. |
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