The National Hemophilia Foundation (NHF) today, in conjunction with the American Kidney Fund (AKF), Arthritis Foundation, and American Autoimmune Related Diseases Association (AARDA), released findings from a new national online survey of patients…
Objective:
The completed pediatric phase 3 pathfinder 5 trial assessed the safety and efficacy of N8-GP (turoctocog alfa pegol, ESPEROCT®) use for routine prophylaxis and treatment of breakthrough bleeds in previously treated…
His project aims to directly address current limitations of hemophilia A gene and protein therapy by the identification and characterization of new hyperactive factor VIII variants based on his previous studies of hyperactive factor IX variants. He…
The recent American Society of Gene & Cell Therapy (ASGCT) Virtual Meeting featured updates from the phase III HOPE-B clinical trial of etranacogene dezaparvovec, an investigational gene therapy developed by uniQure for patients with severe…
BioMarin recently reported key findings from preclinical studies of their investigational, gene therapy Roctavian (valoctocogene roxaparvovec), which was developed for the treatment of adults with severe hemophilia A. The findings were presented at…
Using Math to Understand Bleeding in Hemophilia Patients
Speaker: Karin Leiderman, MD
Did you miss the June 2nd Wednesday Webinar? If so you can listen here to Karin Leiderman, MD discuss Using Math to Understand Bleeding in…
Objective:
Daily administrations of FVIII products are considered useful for providing high FVIII coverage for active patients with hemophilia A. This analysis was performed to determine the daily dose levels required of N8-GP (…
Danger Signals and Inhibitor Development
Speaker: Radoslaw Kaczmarek
Did you miss the June 9th Wednesday Webinar series? If so you can listen here to Redek Kaczmarek, PhD discuss Danger Signals and Inhibitor Development…
The Current Status of Gene Therapy in Hemophilia
Speaker: John Pasi, MB, ChB
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