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Margareth Castro Ozelo
Margareth Castro Ozelo
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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
CS Manno, GF Pierce, VR Arruda, B Glader, M Ragni, JJE Rasko, ...
Nature medicine 12 (3), 342-347, 2006
23552006
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
CS Manno, AJ Chew, S Hutchison, PJ Larson, RW Herzog, VR Arruda, ...
Blood, The Journal of the American Society of Hematology 101 (8), 2963-2972, 2003
8972003
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B
JS Powell, KJ Pasi, MV Ragni, MC Ozelo, LA Valentino, JN Mahlangu, ...
New England Journal of Medicine 369 (24), 2313-2323, 2013
3762013
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B
H Jiang, GF Pierce, MC Ozelo, EV De Paula, JA Vargas, P Smith, ...
Molecular Therapy 14 (3), 452-455, 2006
2462006
An inherited mutation leading to production of only the short isoform of GATA-1 is associated with impaired erythropoiesis
LM Hollanda, CSP Lima, AF Cunha, DM Albuquerque, J Vassallo, ...
Nature genetics 38 (7), 807-812, 2006
2122006
Valoctocogene roxaparvovec gene therapy for hemophilia A
MC Ozelo, J Mahlangu, KJ Pasi, A Giermasz, AD Leavitt, M Laffan, ...
New England Journal of Medicine 386 (11), 1013-1025, 2022
2032022
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
JD Finn, MC Ozelo, DE Sabatino, HWG Franck, EP Merricks, JM Crudele, ...
Blood, The Journal of the American Society of Hematology 116 (26), 5842-5848, 2010
2032010
ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease
NT Connell, VH Flood, R Brignardello-Petersen, R Abdul-Kadir, ...
Blood advances 5 (1), 301-325, 2021
1892021
Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B
LA George, MV Ragni, JEJ Rasko, LJ Raffini, BJ Samelson-Jones, ...
Molecular Therapy 28 (9), 2073-2082, 2020
1402020
Results from a large multinational clinical trial (guardian™ 1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A …
SR Lentz, M Misgav, M Ozelo, SZ Šalek, D Veljkovic, M Recht, ...
Haemophilia 19 (5), 691-697, 2013
1012013
Family history as a risk factor for venous thromboembolism
S Noboa, G Le Gal, K Lacut, B Mercier, C Leroyer, E Nowak, D Mottier, ...
Thrombosis research 122 (5), 624-629, 2008
962008
Inherited risk factors for thrombophilia among children with Legg-Calvé-Perthes disease
VR Arruda, WD Belangero, MC Ozelo, GB Oliveira, RG Pagnano, ...
Journal of Pediatric Orthopaedics 19 (1), 84-87, 1999
961999
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A
H Matsui, C Hegadorn, M Ozelo, E Burnett, A Tuttle, A Labelle, PB McCray, ...
Molecular therapy 19 (4), 723-730, 2011
872011
Results from a large multinational clinical trial (guardian™ 3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety …
R Kulkarni, FA Karim, S Glamocanin, D Janic, V Vdovin, M Ozelo, ...
Haemophilia 19 (5), 698-705, 2013
782013
Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective
K Fischer, P Poonnoose, AL Dunn, P Babyn, MJ Manco‐Johnson, ...
Haemophilia 23 (1), 11-24, 2017
762017
Key issues in inhibitor management in patients with haemophilia
K Gomez, R Klamroth, J Mahlangu, ME Mancuso, ME Mingot, MC Ozelo
Blood Transfusion 12 (Suppl 1), s319, 2014
762014
Frequency of platelet type versus type 2B von Willebrand disease
A Hamilton, M Ozelo, J Leggo, C Notley, H Brown, JP Frontroth, ...
Thrombosis and haemostasis 105 (03), 501-508, 2011
722011
When and how to start prophylaxis in boys with severe hemophilia without inhibitors: communication from the SSC of the ISTH
K Fischer, PW Collins, MC Ozelo, A Srivastava, G Young, VS Blanchette
Journal of Thrombosis and Haemostasis 14 (5), 1105-1109, 2016
712016
Evaluation of the utility of the ISTH‐BAT in haemophilia carriers: a multinational study
PD James, J Mahlangu, C Bidlingmaier, ME Mingot‐Castellano, M Chitlur, ...
Haemophilia 22 (6), 912-918, 2016
692016
Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A
J Mahlangu, R Kaczmarek, A Von Drygalski, S Shapiro, SC Chou, ...
New England Journal of Medicine 388 (8), 694-705, 2023
662023
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