Nina Mitchell
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View article: Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A Open
Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people with severe hemophilia A. Molecular mechanisms underlying sust…
View article: Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A
Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A Open
Introduction Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A. Aim To report safety, tolerability, efficacy, a…
View article: Late breaking Abstracts
Late breaking Abstracts Open
Background: Damoctocog alfa pegol (BAY 94-9027, Jivi ® ) and rurioctocog alfa pegol (BAX 855, Adynovi ® ) are PEGylated, extended half-life, recombinant factor VIII (FVIII) products, indicated for use in patients ≥ 12 years of age with hae…
View article: Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A Open
Gene therapy with AAV5-hFVIII-SQ vector in participants with hemophilia A resulted in sustained, clinically relevant benefit, as measured by a substantial reduction in annualized rates of bleeding events and complete cessation of prophylac…