Tag: Hemophilia
Concizumab May Be Effective Prophylaxis for Hemophilia A or B With Inhibitors
Overall median annualized bleeding rate was 0 with concizumab
FDA Approves First Gene Therapy for Severe Hemophilia A
Roctavian is a viral vector that carries the gene for factor VIII
Fitusiran Beneficial in Hemophilia A, B With or Without Inhibitors
Reduction in annualized bleeding rate seen compared with bypassing agents on demand and on-demand clotting factor concentrates
Benefits of Valoctocogene Roxaparvovec Persist in Hemophilia A
The mean annualized treated bleeding rate decreased by 84.5 percent from baseline to 104 weeks
Gene Therapy Beneficial for Patients With Hemophilia B
One infusion of etranacogene dezaparvovec noninferior and superior to factor IX prophylaxis
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
