Self reported physical activity not significantly different across different severities of hemophilia
1. In this cohort of individuals with hemophilia, the physical activity levels and step count did not significantly differ between ...
Tag: hemophilia
#VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis
Click here to read this study in NEJM. ©2024 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced ...
Concizumab reduces bleeding episodes in hemophilia patients with inhibitors
1. In this phase three open-label randomized trial, concizumab prophylaxis reduced the annualized bleeding rate in patients with hemophilia with ...
#VisualAbstract: 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. ...
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. ...
Etranacogene dezaparvovec gene therapy reduces bleeding in severe hemophilia B
1. Etranacogene dezaparvovec gene therapy was more effective than prophylactic factor IX infusion at reducing annualized bleeding rate amongst patients ...
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, ...
Recombinant viral vector treatment sustains endogenous factor IX activity in patients with hemophilia B
1. A one-time infusion of a recombinant viral vector with a highly expressed factor IX gene resulted in sustained endogenous ...