Search details
1.
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
N Engl J Med
; 388(4): 310-318, 2023 01 26.
Article
in English
| MEDLINE | ID: mdl-36720133
2.
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Blood
; 143(9): 796-806, 2024 Feb 29.
Article
in English
| MEDLINE | ID: mdl-37871576
3.
Inherited causes of clonal haematopoiesis in 97,691 whole genomes.
Nature
; 586(7831): 763-768, 2020 10.
Article
in English
| MEDLINE | ID: mdl-33057201
4.
Extent to which array genotyping and imputation with large reference panels approximate deep whole-genome sequencing.
Am J Hum Genet
; 109(9): 1653-1666, 2022 09 01.
Article
in English
| MEDLINE | ID: mdl-35981533
5.
How I approach bleeding in hospitalized patients.
Blood
; 142(9): 761-768, 2023 08 31.
Article
in English
| MEDLINE | ID: mdl-36652635
6.
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia.
Gene Ther
; 31(5-6): 273-284, 2024 05.
Article
in English
| MEDLINE | ID: mdl-38355967
7.
The importance and evolution of bleeding disorder registries.
Haemophilia
; 30 Suppl 3: 21-28, 2024 Apr.
Article
in English
| MEDLINE | ID: mdl-38571362
8.
A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.
Haemophilia
; 30(1): 214-223, 2024 Jan.
Article
in English
| MEDLINE | ID: mdl-37902390
9.
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.
N Engl J Med
; 383(11): 1018-1027, 2020 09 10.
Article
in English
| MEDLINE | ID: mdl-32905674
10.
Cryptic resolution sites in the vector plasmid lead to the heterogeneities in the rAAV vectors.
J Med Virol
; 95(1): e28433, 2023 01.
Article
in English
| MEDLINE | ID: mdl-36571262
11.
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Blood
; 137(6): 763-774, 2021 02 11.
Article
in English
| MEDLINE | ID: mdl-33067633
12.
Association of factor expression levels with annual bleeding rate in people with haemophilia B.
Haemophilia
; 29(1): 115-122, 2023 Jan.
Article
in English
| MEDLINE | ID: mdl-36331904
13.
Depression and anxiety in persons with Von Willebrand disease.
Haemophilia
; 29(2): 545-554, 2023 Mar.
Article
in English
| MEDLINE | ID: mdl-36516311
14.
Phenotypic analysis of erythrocytes in sickle cell disease using imaging flow cytometry.
Cytometry A
; 101(5): 448-457, 2022 05.
Article
in English
| MEDLINE | ID: mdl-35099119
15.
Author Correction: Inherited causes of clonal haematopoiesis in 97,691 whole genomes.
Nature
; 591(7851): E27, 2021 Mar.
Article
in English
| MEDLINE | ID: mdl-33707633
16.
How do I facilitate a rapid response to a public health emergency requiring plasma collection with a public-private partnership?
Transfusion
; 61(10): 2814-2824, 2021 10.
Article
in English
| MEDLINE | ID: mdl-34510475
17.
The critical need for postmarketing surveillance in gene therapy for haemophilia.
Haemophilia
; 27 Suppl 3: 126-131, 2021 Feb.
Article
in English
| MEDLINE | ID: mdl-32495492
18.
Urinary cross-linked carboxyterminal telopeptide, a bone resorption marker, decreases after vaso-occlusive crises in adults with sickle cell disease.
Blood Cells Mol Dis
; 80: 102369, 2020 02.
Article
in English
| MEDLINE | ID: mdl-31677454
19.
Long-term safety and efficacy results from the phase 3b, open-label, multicentre Continuation study of rurioctocog alfa pegol for prophylaxis in previously treated patients with severe haemophilia A.
Haemophilia
; 26(4): e168-e178, 2020 Jul.
Article
in English
| MEDLINE | ID: mdl-32597029
20.
Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study.
Haemophilia
; 26(3): 494-502, 2020 May.
Article
in English
| MEDLINE | ID: mdl-32227570