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Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1.
O'Mahony, Brian; Dunn, Amy L; Leavitt, Andrew D; Peyvandi, Flora; Ozelo, Margareth C; Mahlangu, Johnny; Peerlinck, Kathelijne; Wang, Jiaan-Der; Lowe, Gillian C; Tan, Chee Wee; Giermasz, Adam; Tran, Huyen; Khoo, Teh-Liane; Cockrell, Erin; Pepperell, Dominic; Chambost, Hervé; López Fernández, Maria Fernanda; Kazmi, Rashid; Majerus, Elaine; Skinner, Mark W; Klamroth, Robert; Quinn, Jennifer; Yu, Hua; Wong, Wing Yen; Robinson, Tara M; Pipe, Steven W.
Afiliação
  • O'Mahony B; Irish Haemophilia Society, Dublin, Ireland; Trinity College, Dublin, Ireland. Electronic address: brian@haemophilia.ie.
  • Dunn AL; The Division of Hematology, Oncology, and BMT at Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, Ohio, USA.
  • Leavitt AD; University of California San Francisco, San Francisco, California, USA.
  • Peyvandi F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy.
  • Ozelo MC; Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil.
  • Mahlangu J; Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa.
  • Peerlinck K; Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium.
  • Wang JD; Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan.
  • Lowe GC; West Midlands Comprehensive Care Haemophilia Centre, Queen Elizabeth Hospital, Birmingham, UK.
  • Tan CW; Department of Haematology, Royal Adelaide Hospital, Adelaide, South Australia, Australia; University of Adelaide, Adelaide, South Australia, Australia.
  • Giermasz A; Hemophilia Treatment Center, University of California Davis, Sacramento, California, USA.
  • Tran H; Haemostasis & Thrombosis Unit, Haemophilia Treatment Centre, The Alfred Hospital, Melbourne, Victoria, Australia.
  • Khoo TL; Institute of Haematology, Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia.
  • Cockrell E; Pediatric Hematology Oncology, Saint Joseph's Children's Hospital, Tampa, Florida, USA.
  • Pepperell D; Department of Haematology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia.
  • Chambost H; AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France.
  • López Fernández MF; Complejo Hospitalario Universitario A Coruña, A Coruña, Spain.
  • Kazmi R; Department of Haematology, Southampton University Hospital, Southampton, UK.
  • Majerus E; Department of Medicine, Washington University in St. Louis, St. Louis, Missouri, USA.
  • Skinner MW; Institute for Policy Advancement Ltd, Washington, DC, USA; McMaster University, Hamilton, Ontario, Canada.
  • Klamroth R; Comprehensive Care Haemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, Germany; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Germany.
  • Quinn J; BioMarin Pharmaceuticals UK Ltd, London, UK.
  • Yu H; BioMarin Pharmaceutical Inc, Novato, California, USA.
  • Wong WY; BioMarin Pharmaceutical Inc, Novato, California, USA.
  • Robinson TM; BioMarin Pharmaceutical Inc, Novato, California, USA.
  • Pipe SW; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.
J Thromb Haemost ; 21(12): 3450-3462, 2023 12.
Article em En | MEDLINE | ID: mdl-37678546
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hemofilia A Limite: Adult / Humans / Male Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hemofilia A Limite: Adult / Humans / Male Idioma: En Ano de publicação: 2023 Tipo de documento: Article