Tuesday, December 5, 2023
Clinical Advances in
After many years, gene therapy in patients with hemophilia has started to show promising results in alleviating symptoms and improving the disease phenotype. This collection of programs provides important information on the science behind gene therapy for hemophilia, the latest safety and efficacy data from clinical trials, and the potential role for gene therapy in addressing the challenges of current treatment methods.
Supported by an independent educational grant from
BioMarin
Presented through a collaboration between Medscape and
Supported by an independent educational grant from
BioMarin
Presented through a collaboration between Medscape and
Supported by an independent educational grant from
BioMarin
Presented through a collaboration between Medscape and
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Steering Committee Chair
Professor of Haemostasis and Thrombosis, Barts and the London School of Medicine and Dentistry, London, United Kingdom
Professor of Haematology, Faculty of Health Sciences, Department of Molecular Medicine and Haematology, University of the Witwatersrand; Head of Clinical Haematology Ward and Service, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa
Assistant Professor; Director, IHTC Hemocentro UNICAMP, University of Campinas; Director, WFH International Hemophilia Training Center, Sao Paulo, Brazil
Professor of Internal Medicine, University of Milan; Director, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Professor of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, United States
Associate Member; Director of Clinical Hematology; Director, Pediatric Hemophilia Treatment Center, Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, United States
Steering Committee Chair
Has disclosed the following relevant financial relationships
Served as an advisor or consultant for: Alnylam Pharmaceuticals, Inc; Biogen; BioMarin Pharmaceutical Inc.; Octapharma; Roche; Shire; Swedish Orphan Biovitrum
Served as a speaker or a member of a speakers bureau for: Bayer HealthCare Pharmaceuticals; Biotest; Novo Nordisk; Pfizer Inc
Has disclosed the following relevant financial relationships
Served as an advisor or consultant for: Amgen Inc.; Baxalta; Bayer HealthCare; Biogen; Biotest; CSL Behring; Catalyst Pharmaceutical Partners, Inc.; Novo Nordisk; Roche; Spark
Served as a speaker or a member of a speakers bureau for: Alnylam Pharmaceuticals, Inc; Bayer HealthCare; Biogen; Biotest; Novo Nordisk; Pfizer Inc.; Swedish Orphan Biovitrum; Roche; Shire
Received grants for clinical research from: Bayer HealthCare; Biogen; BioMarin Pharmaceutical Inc.; CSL Behring; Novo Nordisk; Swedish Orphan Biovitrum; Roche; Unique Pharmaceuticals
Has disclosed the following relevant financial relationships
Served as an advisor or consultant for: BioMarin Pharmaceutical Inc.; CSL Behring; Novo Nordisk; Pfizer Inc.; Roche; Shire
Served as a speaker or a member of a speakers bureau for: BioMarin Pharmaceutical Inc.; Grifols; Novo Nordisk; Pfizer Inc.; Roche; Shire
Received grants for clinical research from: Bioverativ; Pfizer Inc.; Shire
Has disclosed the following relevant financial relationships
Served as an advisor or consultant for: Kedrion Biopharma Inc.; LFB SA
Served as a speaker or a member of a speakers bureau for: Ablynx; Alnylam Pharmaceuticals, Inc; F. Hoffmann-La Roche; Grifols; Shire; Swedish Orphan Biovitrum
Other: Advisory board member for Ablynx; F. Hoffmann-La Roche; Shire
Has disclosed the following relevant financial relationships
Served as an advisor or consultant for: Alnylam Pharmaceuticals, Inc; Bayer HealthCare; BioMarin Pharmaceutical Inc.; Bioverativ; CSL Behring; Novo Nordisk; Pfizer Inc.; Roche; Spark; UniQure
Received grants for clinical research from: Pfizer Inc.; Siemens Healthcare Diagnostics Inc.; Shire
Has disclosed no relevant financial relationships