An exploration of why men with severe haemophilia might not want gene therapy: The exigency study
Speaker(s): Simon Fletcher
NHF's Wednesday Webinars are a free education series open to providers and…
As investigational hemophilia gene therapies begin receiving regulatory approvals, community stakeholders have recognized the acute need for people with hemophilia (PWH) and healthcare professionals (HCPs) to be fully engaged in shared decision…
Dr. Jill Johnsen is scientist and physician at the Washington Center for Bleeding Disorders in Seattle, WA. She is an Associate Member at the Bloodworks Research Institute and also an Associate Professor of Medicine in the Division of…
Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for REBINYN®, the company’s recombinant, GlycoPEGylated extended half-life therapy. The approval is…
BioMarin recently shared findings from a phase 3 clinical trial (GENEr8-1) evaluating the presence of vector DNA in patients two years after they received a single dose of valoctocogene roxaparvovec, the company’s investigational hemophilia A gene…
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Dr. Andrew Yee is an assistant professor of pediatrics within the Department of Pediatrics, Hematology-Oncology Section at Baylor College of Medicine. Dr. Yee earned his doctorate from Rice University where he studied the biological responses of…
You are cordially invited to participate in a Meet-Up Event for people affected by ultra-rare inherited bleeding disorders (URBDs) being held on September 9 – 11 at the Marriott SW, in Burr Ridge, Illinois. We know that you face significant…
MSKUS
Speaker(s): Peter Aguero
NHF's Wednesday Webinars are a free education series open to providers and community members. Register to attend and learn about the latest in research, breakthrough developments, and more.…