The activity launched on Madata through Jare presented, but data collection is ongoing. Respondent confidentiality was maintained and responses were de-identified and aggregated prior to analyses. The survey instrument was made available online to physicians without monetary compensation or charge. METHODS: A continuing medical education (CME)-certified clinical practice assessment comprising 25 multiple choice questions that measured knowledge, attitudes, and perspectives about gene therapy was developed. The objective of the current study is to assess clinicians' needs with regard to understanding the science and mechanism for gene therapy, the different technologies being proposed, identifying candidates for gene therapy, disease areas for which gene therapy is being explored, and the latest data on the potential role of gene therapy in hemophilia A and B. To identify and address practice gaps among healthcare providers who manage patients with hemophilia, a collaboration between the National Hemophilia Foundation (NHF), European Haemophilia Consortium (EHC), World Federation of Hemophilia (WFH), and Medscape Education was established. Gene therapy, by contrast, offers the hope of a functional cure through endogenous expression of the factor VIII or factor IX genes. BACKGROUND: Currently available treatments for hemophilia A and B require frequent intravenous infusions.
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