The recent Congress of the International Society on Thrombosis and Haemostasis (ISTH) included some noteworthy findings on the rare blood disorders news front. Hemab Therapeutics, which specializes in developing therapies that target serious…
Sanofi recently shared new clinical trial updates for their investigational hemophilia therapy fitusiran. The Phase 3 ATLAS-PPX study is evaluating the efficacy and safety of fitusiran (80 mg) when administered prophylactically (once per month) to…
Markers of autoimmunity in immune thrombocytopenia
Speaker(s): Dr. Marie Hollenhorst
NHF's Wednesday Webinars are a free education series open to providers and community members. Register to attend and learn…
50 Years of the JGP Research Fellowship
Speaker(s): NHF Research Team
NHF's Wednesday Webinars are a free education series open to providers and community members. Register to attend and learn about the latest…
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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