Tag: Hemophilia
Once-Weekly Efanesoctocog Alfa Beneficial in Severe Hemophilia A
Superior bleeding prevention to prestudy prophylaxis, normal or near-normal factor VIII activity, improvements in physical health, pain
FDA Approves First Gene Therapy for Adults With Hemophilia B
Benefits include decreased need for routine factor IX replacement prophylaxis and reduction in annualized bleeding rate
AAV Gene Transfer Feasible for the Treatment of Hemophilia A
In a phase 1-2 trial, 16 of 18 men had sustained expression of factor VIII after gene transfer with adeno-associated viral vector (SPK-8011)
1,125,000 Men/Boys Expected to Have Hemophilia Worldwide
Prevalence of all severities of hemophilia A and hemophilia B estimated at 17.1 and 3.8 cases per 100,000
Emicizumab Prophylaxis Cuts Bleeding in Hemophilia A
Lower bleeding rate with administration once weekly, every two weeks versus no prophylaxis
ASH: High-Dose Gene Transfer Beneficial in Severe Hemophilia A
Factor VIII gene transfer tied to sustained normalization of factor VIII activity levels
Gene Therapy Shows Beneficial Effect in Men With Hemophilia B
Reduction in annualized bleeding rate and in factor use with infusion of viral vector with transgene
FDA OKs Drug for Hemophilia A With Factor VIII Inhibitors
Weekly prophylactic treatment associated with substantial decrease in bleeding episodes
Prophylaxis Linked to Improved Function, HRQoL in Hemophilia
Improved function, QoL, activity, pain but not joint structure in adults with hemophilia with joint disease
ASH: Single Dose of Gene Therapy Effective in Hemophilia B
Small, preliminary trial suggests it may free hemophilia B patients from transfusions
