Tag: Hemophilia
FDA Approves Hympavzi for Hemophilia
Approval is for prophylactic treatment and targets protein in the blood clotting process
Fidanacogene Elaparvovec Superior to Prophylaxis for Hemophilia B
Adeno-associated virus gene-therapy vector for hemophilia B superior for reducing annualized bleeding rate
Efanesoctocog Alfa Prophylaxis Beneficial for Children With Hemophilia A
Authors note high sustained factor VIII activity and protection against bleeding episodes, including bleeding into joints
ISTH Issues Clinical Practice Guideline for the Treatment of Hemophilia
Prophylaxis recommended over episodic treatment of bleeding events for severe and moderately severe hemophilia A and B
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
