Activated factor VIII-mimetic prophylaxis associated with reduced bleeding in hemophilia A
1. In adolescents and adults with hemophilia A, subcutaneous Mim8 prophylaxis reduced treated bleeding events versus on-demand therapy and versus ...
Tag: hemophilia A
Pfizer’s First Of Its Kind Gene Therapy Shines In Phase 3 Trials For Treatment of Hemophilia A
Giroctocogene fitelparvovec, a novel gene therapy, demonstrated superiority to routine therapy in treating hemophilia A. Giroctocogene fitelparvovec resulted in a ...
Efanesoctocog alfa in children with hemophilia A reduces bleeding
1. In this randomized controlled trial, a prophylactic weekly dose of efanesoctocog alfa (ALTUVIIIO) resulted in sustained factor VIII activity ...
#VisualAbstract: Phase 3 Trial of Concizumab in Hemophilia with Inhibitors
Click to view this study in NEJM. ©2023 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced ...
Once-a-month fitusiran reduces bleeding rates in patients with hemophilia A and B with inhibitors
1. Annualized bleeding rate was reduced by 91% in the fitusiran prophylaxis group compared to the bypassing agents on-demand group. ...
Valactogene roxaparvovec for hemophilia A demonstrates factor VIII durability for two years
1. At least two years after gene transfer, valoctocogene roxaparvovec demonstrated durability of factor VIII activity and bleeding reduction in ...
Efanesoctocog alfa is efficacious in treating severe hemophilia A
1. Efanesoctocog alfa was superior to prestudy factor VIII prophylaxis in preventing bleeding in patients with severe hemophilia A. 2. ...
Valoctocogene roxaparvovec gene therapy efficacious for hemophilia A treatment
1. The adeno-associated virus 5 (AAV5)-based gene therapy, valoctocogene roxaparvovec (AAV5-hFVIII-SQ), resulted in sustained factor VIII production, reduced bleeding rates, ...
