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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

Researchers

Presenter

  • Brian O'Mahony

Principal Investigators

  • Kathelijne Peerlinck

  • Jiaan-Der Wang

  • Gillian Lowe

  • Chee Wee Tan

  • Adam Giermasz

  • Huyen A Tran

  • Teh-Liane Khoo

  • Erin Cockrell

  • Dominic Pepperell

  • Herve Chambost

  • Maria Fernanda Lopez Fernandez

  • Rashid Kazmi

  • Elaine Majerus

  • Mark W Skinner

  • Robert Klamroth

  • Jennifer Quinn

  • Hua Yu

  • Wing Yen Wong

  • Adebayo Lawal

  • Tara M Robinson

  • Benjamin Kim

Medical Centers

  • West Midlands Comprehensive Care Haemophilia Centre, Queen Elizabeth Hospital, Birmingham, United Kingdom

  • Trinity College Dublin, Dublin, Ireland

  • Pediatric Hematology Oncology, St. Joseph's Children's Hospital, Tampa, FL

  • Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa

  • Department of Haematology, Southampton University Hospital, Southampton, United Kingdom

  • Irish Haemophilia Society, Dublin, Ireland

  • Department of Haematology, Fiona Stanley Hospital, Murdoch, Australia

  • Haemostasis & Thrombosis Unit, Haemophilia Treatment Centre, The Alfred Hospital, Melbourne, Australia

  • Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan

  • Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium

  • Hemophilia Treatment Center, University of California Davis, Sacramento, CA

  • Institute of Haematology, Royal Prince Alfred Hospital, Camperdown, Australia

  • AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone, Aix Marseille University, INSERM, INRA, CVN, Marseille, France

  • Department of Haematology, Royal Adelaide Hospital, Adelaide, Australia

  • McMaster University, Hamilton, Canada

  • Complejo Hospitalario Universitario A Coruña, A Coruña, Spain

  • Comprehensive Care Haemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, Germany

  • Department of Medicine, Washington University, St. Louis, MO

Locations

  • United Kingdom

  • Belgium

  • Ireland

  • United States

  • Australia

  • South Africa

  • France

  • Canada

  • Spain

  • Germany

  • Taiwan

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

  • Pulmonary/Respiratory Diseases

  • Central Nervous System (CNS)

Disease

  • Hemophilia

  • Depression

  • Anxiety

  • Chronic obstructive pulmonary disease

Biomarkers

  • N/A

Drug/Treatment

  • FVIII

Outcome

  • N/A

Study Design

  • N/A

Phase

  • III

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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