selected publications
- Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations. Pediatric blood & cancer. 2024 Review GET IT
- Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations. Pediatric blood & cancer. 2024 Review GET IT
- Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database. Blood advances. 2024 Academic Article GET IT
- A review of the pharmacokinetics, efficacy and safety of high-purity factor X for the prophylactic treatment of hereditary factor X deficiency. Haemophilia : the official journal of the World Federation of Hemophilia. 2022 Review GET IT
- Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain. Haemophilia : the official journal of the World Federation of Hemophilia. 2022 Academic Article GET IT
- Haemophilia: factoring in new therapies. British journal of haematology. 2021 Information Resource GET IT
- Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural). Haemophilia : the official journal of the World Federation of Hemophilia. 2020 Academic Article GET IT
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The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development.
Blood advances.
2020
Academic Article
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Times cited: 26 -
Use of plasma-derived factor X concentrate in neonates and infants with congenital factor X deficiency.
Journal of thrombosis and haemostasis : JTH.
2020
Academic Article
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Times cited: 2 -
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 : the official journal of the World Federation of Hemophilia.
2020
Academic Article
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Times cited: 2 -
Immunophenotypic, cytotoxic, proteomic and genomic characterization of human cord blood vs. peripheral blood CD56Dim NK cells.
Innate immunity.
2019
Academic Article
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Times cited: 13 -
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
The New England journal of medicine.
2017
Academic Article
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Times cited: 555 -
Radionuclide synovectomy/synoviorthesis (RS) in patients with bleeding disorders: A review of patient and procedure demographics and functional outcomes in the ATHNdataset.
Haemophilia : the official journal of the World Federation of Hemophilia.
2017
Academic Article
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Times cited: 4 -
Radionuclide synovectomy/synoviorthesis (RS) in persons with bleeding disorders: A review of impact of national guidance on frequency of RS using the ATHNdataset.
Haemophilia : the official journal of the World Federation of Hemophilia.
2017
Article
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Times cited: 3 -
Pilot trial of risk-adapted cyclophosphamide intensity based conditioning and HLA matched sibling and unrelated cord blood stem cell transplantation in newly diagnosed pediatric and adolescent recipients with acquired severe aplastic anemia.
Pediatric blood & cancer.
2014
Academic Article
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Times cited: 6 -
Promoter methylation-mediated inactivation of PCDH10 in acute lymphoblastic leukemia contributes to chemotherapy resistance.
Genes, chromosomes & cancer.
2011
Academic Article
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Times cited: 28