The phase 3 clinical study of gene transfer in hemophilia A using the BioMarin vector AAV5-hFVIII-SQ (also identified as valoctocogene roxaparvovec) recently reported the results obtained in as many as 134 adult men with severe hemophilia A, so that it is at the moment the largest gene transfer study ever conducted in a rare monogenic disorder [...]
Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFV...
BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therape...
Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clo...
BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, ...
Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attr...
Historically, the standard of care for hemophilia A has been intravenous administration of exogenous...
BACKGROUND Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, s...
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. T...
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. T...
BACKGROUND: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based ...
Hemophilia A and B are X-linked monogenic disorders caused by deficiencies in coagulation factor VII...
The last two decades has seen significant progress in the treatment of hemophilia A. The developmen...
Factor IX deficiency (hemophilia B) is less common than factor VIII deficiency (hemophilia A) and in...
In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are curren...
In the last decade, enormous progress has been made in the development of gene therapy for hemophili...
Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFV...
BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therape...
Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clo...
BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, ...
Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attr...
Historically, the standard of care for hemophilia A has been intravenous administration of exogenous...
BACKGROUND Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, s...
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. T...
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. T...
BACKGROUND: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based ...
Hemophilia A and B are X-linked monogenic disorders caused by deficiencies in coagulation factor VII...
The last two decades has seen significant progress in the treatment of hemophilia A. The developmen...
Factor IX deficiency (hemophilia B) is less common than factor VIII deficiency (hemophilia A) and in...
In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are curren...
In the last decade, enormous progress has been made in the development of gene therapy for hemophili...
Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFV...
BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therape...
Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clo...
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