Search | VHL Search Portal

1.

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.

Mahlangu, Johnny; Kaczmarek, Radoslaw; von Drygalski, Annette; Shapiro, Susan; Chou, Sheng-Chieh; Ozelo, Margareth C; Kenet, Gili; Peyvandi, Flora; Wang, Michael; Madan, Bella; Key, Nigel S; Laffan, Michael; Dunn, Amy L; Mason, Jane; Quon, Doris V; Symington, Emily; Leavitt, Andrew D; Oldenburg, Johannes; Chambost, Hervé; Reding, Mark T; Jayaram, Kala; Yu, Hua; Mahajan, Reena; Chavele, Konstantia-Maria; Reddy, Divya B; Henshaw, Joshua; Robinson, Tara M; Wong, Wing Yen; Pipe, Steven W.

N Engl J Med ; 388(8): 694-705, 2023 02 23.

Article in English | MEDLINE | ID: mdl-36812433

2.

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

Ozelo, Margareth C; Mahlangu, Johnny; Pasi, K John; Giermasz, Adam; Leavitt, Andrew D; Laffan, Michael; Symington, Emily; Quon, Doris V; Wang, Jiaan-Der; Peerlinck, Kathelijne; Pipe, Steven W; Madan, Bella; Key, Nigel S; Pierce, Glenn F; O'Mahony, Brian; Kaczmarek, Radoslaw; Henshaw, Joshua; Lawal, Adebayo; Jayaram, Kala; Huang, Mei; Yang, Xinqun; Wong, Wing Y; Kim, Benjamin.

N Engl J Med ; 386(11): 1013-1025, 2022 03 17.

Article in English | MEDLINE | ID: mdl-35294811

3.

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results.

Königs, Christoph; Ozelo, Margareth C; Dunn, Amy; Kulkarni, Roshni; Nolan, Beatrice; Brown, Simon A; Schiavulli, Michele; Gunawardena, Sriya; Mukhopadhyay, Sutirtha; Jayawardene, Deepthi; Winding, Bent; Carcao, Manuel.

Blood ; 139(26): 3699-3707, 2022 06 30.

Article in English | MEDLINE | ID: mdl-35421219

4.

Comparative transcriptomic analysis of circulating endothelial cells in sickle cell stroke.

de Castro, Júlia Nicoliello Pereira; da Silva Costa, Sueli Matilde; Camargo, Ana Carolina Lima; Ito, Mirta Tomie; de Souza, Bruno Batista; de Haidar E Bertozzo, Victor; Rodrigues, Thiago Adalton Rosa; Lanaro, Carolina; de Albuquerque, Dulcinéia Martins; Saez, Roberta Casagrande; Saad, Sara Teresinha Olalla; Ozelo, Margareth Castro; Cendes, Fernando; Costa, Fernando Ferreira; de Melo, Mônica Barbosa.

Ann Hematol ; 103(4): 1167-1179, 2024 Apr.

Article in English | MEDLINE | ID: mdl-38386032

5.

Current and emerging gene therapies for haemophilia A and B.

Kaczmarek, Radoslaw; Miesbach, Wolfgang; Ozelo, Margareth C; Chowdary, Pratima.

Haemophilia ; 30 Suppl 3: 12-20, 2024 Apr.

Article in English | MEDLINE | ID: mdl-38528615

6.

Promoting pain coping skills in haemophilia: A remote intervention integrating exercise and pain education.

Feldberg, Glenda; Ricciardi, Janaína B S; Zorzi, Alessandro R; Yamaguti-Hayakawa, Gabriela G; Ozelo, Margareth C.

Haemophilia ; 30(3): 800-808, 2024 May.

Article in English | MEDLINE | ID: mdl-38523258

7.

Impaired repair properties of endothelial colony-forming cells in patients with granulomatosis with polyangiitis.

Del Rio, Ana Paula Toledo; Frade-Guanaes, Jéssica O; Ospina-Prieto, Stephanie; Duarte, Bruno K L; Bertolo, Manoel Barros; Ozelo, Margareth C; Sachetto, Zoraida.

J Cell Mol Med ; 26(19): 5044-5053, 2022 10.

Article in English | MEDLINE | ID: mdl-36052734

8.

Haemophilia gene therapy-Update on new country initiatives.

Reiss, Ulrike M; Mahlangu, Johnny; Ohmori, Tsukasa; Ozelo, Margareth C; Srivastava, Alok; Zhang, Lei.

Haemophilia ; 28 Suppl 4: 61-67, 2022 May.

Article in English | MEDLINE | ID: mdl-35521726

9.

Aquatic exercise in patients with haemophilia: Electromyographic and functional results from a prospective cohort study.

Feldberg, Glenda; Ricciardi, Janaína B S; Zorzi, Alessandro R; Colella, Marina P; Ozelo, Margareth C.

Haemophilia ; 27(2): e221-e229, 2021 Mar.

Article in English | MEDLINE | ID: mdl-33595163

10.

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

George, Lindsey A; Ragni, Margaret V; Rasko, John E J; Raffini, Leslie J; Samelson-Jones, Benjamin J; Ozelo, Margareth; Hazbon, Maria; Runowski, Alexa R; Wellman, Jennifer A; Wachtel, Katie; Chen, Yifeng; Anguela, Xavier M; Kuranda, Klaudia; Mingozzi, Federico; High, Katherine A.

Mol Ther ; 28(9): 2073-2082, 2020 09 02.

Article in English | MEDLINE | ID: mdl-32559433

11.

Gene Therapy for Hemophilia A. Reply.

Ozelo, Margareth C; Wong, Wing Y.

N Engl J Med ; 386(23): 2247, 2022 06 09.

Article in English | MEDLINE | ID: mdl-35675187

12.

Long-term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B-YOND extension study.

Pasi, K John; Fischer, Kathelijn; Ragni, Margaret; Kulkarni, Roshni; Ozelo, Margareth C; Mahlangu, Johnny; Shapiro, Amy; P'Ng, Stephanie; Chambost, Hervé; Nolan, Beatrice; Bennett, Carolyn; Matsushita, Tadashi; Winding, Bent; Fruebis, Joachim; Yuan, Huixing; Rudin, Dan; Oldenburg, Johannes.

Haemophilia ; 26(6): e262-e271, 2020 Nov.

Article in English | MEDLINE | ID: mdl-32497409

13.

Longitudinal sequencing of RUNX1 familial platelet disorder: new insights into genetic mechanisms of transformation to myeloid malignancies.

Duarte, Bruno K L; Yamaguti-Hayakawa, Gabriela G; Medina, Samuel S; Siqueira, Lúcia H; Snetsinger, Brooke; Costa, Fernando F; Rauh, Michael J; Ozelo, Margareth C.

Br J Haematol ; 186(5): 724-734, 2019 09.

Article in English | MEDLINE | ID: mdl-31124578

14.

Gene Therapy: Paving New Roads in the Treatment of Hemophilia.

Yamaguti-Hayakawa, Gabriela G; Ozelo, Margareth C.

Semin Thromb Hemost ; 45(7): 743-750, 2019 Oct.

Article in English | MEDLINE | ID: mdl-31096314

15.

Haemophilia Experiences, Results and Opportunities (HERO study) in Brazil: Assessment of the psychosocial effects of haemophilia in patients and caregivers.

Lorenzato, Claudia S; Santos, Rafael B; Fagundes, Gabriel Z Z; Ozelo, Margareth C.

Haemophilia ; 25(4): 640-650, 2019 Jul.

Article in English | MEDLINE | ID: mdl-31115094

16.

First hemophilia B gene therapy approved: More than two decades in the making.

Herzog, Roland W; VandenDriessche, Thierry; Ozelo, Margareth C.

Mol Ther ; 31(1): 1-2, 2023 01 04.

Article in English | MEDLINE | ID: mdl-36528029

17.

Routine clinical care data for population pharmacokinetic modeling: the case for Fanhdi/Alphanate in hemophilia A patients.

Chelle, Pierre; Yeung, Cindy H T; Bonanad, Santiago; Morales Muñoz, Juan Cristóbal; Ozelo, Margareth C; Megías Vericat, Juan Eduardo; Iorio, Alfonso; Spears, Jeffrey; Mir, Roser; Edginton, Andrea.

J Pharmacokinet Pharmacodyn ; 46(5): 427-438, 2019 10.

Article in English | MEDLINE | ID: mdl-31115857

18.

Correction to: Routine clinical care data for population pharmacokinetic modeling: the case for Fanhdi/Alphanate in hemophilia A patients.

Chelle, Pierre; Yeung, Cindy H T; Bonanad, Santiago; Morales Muñoz, Juan Cristóbal; Ozelo, Margareth C; Megías Vericat, Juan Eduardo; Iorio, Alfonso; Spears, Jeffrey; Mir, Roser; Edginton, Andrea.

J Pharmacokinet Pharmacodyn ; 46(5): 439, 2019 Oct.

Article in English | MEDLINE | ID: mdl-31302829

19.

Chronic pain in patients with hemophilia: Influence of kinesiophobia and catastrophizing thoughts.

Feldberg, Glenda; Ricciardi, Janaína B S; Zorzi, Alessandro R; Colella, Marina P; Yamaguti-Hayakawa, Gabriela G; Ozelo, Margareth C.

Haemophilia ; 29(1): 386-389, 2023 Jan.

Article in English | MEDLINE | ID: mdl-36446751

20.

Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial.

Lentz, Steven R; Janic, Dragana; Kavakli, Kaan; Miljic, Predrag; Oldenburg, Johannes; C Ozelo, Margareth; Santagostino, Elena; Suzuki, Takashi; Zupancic Salek, Silva; Korsholm, Lars; Matytsina, Irina; Tiede, Andreas.

Haemophilia ; 24(6): e391-e394, 2018 Nov.

Article in English | MEDLINE | ID: mdl-30402994

VHL Search Portal

Information and Knowledge for Health

Config filters

Your selection (0)

Export format:

Export: