First Gene Therapy for Hemophilia B Gets FDA Approval
The approval was based on data from the HOPE-B trial that included 54 male participants with severe or moderately severe hemophilia B.
Diagnosis & Disease Information
Stable Clinical Outcomes Seen With Octocog Alfa Prophylaxis in Hemophilia A
Researchers sought to determine whether prophylaxis with octocog alfa would lead to stable bleeding rates in patients with hemophilia A.
Non-Alcoholic Fatty Liver Disease Prevalent Among Patients With Hemophilia
Researchers sought to determine the prevalence of NAFLD and associated factors in patients with hemophilia.
FDA Accepts Resubmitted BLA for Valoctocogene Roxaparvovec for Hemophilia A
Valoctocogene roxaparvovec is administered as a single infusion to produce clotting factor VIII.
Rebinyn Approval for Hemophilia B Expanded to Include Routine Prophylaxis
Rebinyn is a recombinant DNA-derived coagulation Factor IX concentrate with an extended half-life.
Millennials With Hemophilia: Attitudes and Expectations From Treatment
Researchers sought to determine the attitudes and expectations of patients with hemophilia belonging to Generation Y regarding their treatment.
Once-Weekly Efanesoctocog Alfa Prevents Bleeding Episodes in Hemophilia A Trial
The phase 3 XTEND-1 study evaluated efanesoctocog alfa in 159 patients with severe hemophilia A who were previously treated with factor VIII replacement therapy.
Durable Response Observed With Gene Therapy for Hemophilia A
The phase 3 GENEr8-1 study evaluated valoctocogene roxaparvovec in adults with severe hemophilia and residual FVIII levels less than or equal to 1 IU/dL.
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