Detalhe da pesquisa
1.
Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.
N Engl J Med
; 389(9): 783-794, 2023 Aug 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-37646676
2.
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
N Engl J Med
; 388(8): 706-718, 2023 02 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-36812434
3.
Gene Therapy for Hemophilia B: Achievements, Open Issues, and Perspectives.
Semin Thromb Hemost
; 2024 May 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-38821066
4.
Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results.
Blood
; 140(2): 89-98, 2022 07 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-35439298
5.
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies.
Haematologica
; 2024 Mar 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-38450517
6.
Outcomes and outcome measures.
Haemophilia
; 30 Suppl 3: 112-119, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38504408
7.
The value-based healthcare approach to haemophilia: Development of outcome measures for the evaluation of care of people with haemophilia.
Haemophilia
; 30(2): 437-448, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38314918
8.
Interim analyses of the multinational real-world prospective cohort HEM-POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A.
Eur J Haematol
; 112(2): 286-295, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-37876046
9.
Pattern of use and clinical outcomes with rIX-FP in pediatric/adolescent patients with haemophilia B in Italy: Results from IDEAL real-world study.
Eur J Haematol
; 112(5): 765-775, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38223989
10.
Translational readthrough at F8 nonsense variants in the factor VIII B domain contributes to residual expression and lowers inhibitor association.
Haematologica
; 108(2): 472-482, 2023 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35924581
11.
Clinical, economic, and health-related quality of life burden associated with von Willebrand disease in adults and children: Systematic and targeted literature reviews.
Haemophilia
; 29(2): 411-422, 2023 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-36097135
12.
Modulation of factor VIII pharmacokinetics by genetic components in factor VIII receptors.
Haemophilia
; 29(2): 479-487, 2023 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-36533781
13.
Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.
Haemophilia
; 29(2): 435-444, 2023 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-36469855
14.
IDEAL study: A real-world assessment of pattern of use and clinical outcomes with recombinant coagulation factor IX albumin fusion protein (rIX-FP) in patients with haemophilia B in Italy.
Haemophilia
; 29(1): 135-144, 2023 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-36423202
15.
Prophylaxis with recombinant von Willebrand factor in patients with type 3 von Willebrand disease: Results of a post hoc analysis from a phase 3 trial.
Eur J Haematol
; 111(1): 29-40, 2023 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-36823994
16.
von Willebrand disease and von Willebrand factor.
Haemophilia
; 28 Suppl 4: 11-17, 2022 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-35521725
17.
Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.
Haemophilia
; 28(6): 1033-1043, 2022 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-35905294
18.
Predictors of inhibitor eradication by primary immune tolerance induction in severe haemophilia A with high responding inhibitors.
Haemophilia
; 28(1): 55-64, 2022 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-34727394
19.
Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.
Haemophilia
; 28(4): 548-556, 2022 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-35475308
20.
Use of the von Willebrand factor concentrate with low factor VIII content to manage patients with inherited von Willebrand disease requiring surgical or secondary long-term prophylaxis: An expert opinion paper from an Italian panel.
Eur J Haematol
; 109(2): 121-128, 2022 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-35531770