The U.S. Food and Drug Administration (FDA) has accepted CSL Behring’s Biologics License Application (BLA) for priority review of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B.
Etranacogene dezaparvovec, also known…
Individuals and families in the bleeding disorders community and those who experience other chronic rare conditions have not typically been engaged in patient reported registries or in clinical studies during the initial study design phase. The more…
The objective of this study was to assess the relationship between self-reported physical activity, treatment regimen, mental health, and pain in persons with hemophilia (PWH) enrolled in CVR. Despite education to the contrary, PWSH continue to…
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In order to become your go-to trusted resource for information on bleeding and blood disorders, NHF is asking for your…
June 3 is the birth date of the late physician and researcher, Dr. Charles Drew. Dr. Drew is remembered for his legacy in creating valuable techniques and processes in blood storage and blood transfusion that are still used today. He is also…
BioMarin recently announced that their planned timeline for the resubmission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for valoctocogene roxaparvovec, also known as Roctavian, has been pushed back to the end…
Paul Monahan is Translational and Clinical Development Lead for Hematology Gene Therapy at Spark Therapeutics. Prior to 2015, Dr. Monahan was Professor of Pediatrics, Hematology/Oncology at the University of North Carolina at Chapel Hill, where he…
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