Gene therapy for hemophilia B allowed almost three-fourths of patients to discontinue prophylactic factor IX therapy with no increase in bleeding, results of the pivotal BENEGENE-2 trial showed. The ...
The FDA on Tuesday approved the first gene therapy for treating hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of factor IX. Etranacogene dezaparvovec ...
Allison Wheeler, MD, delves into the complexities of prophylaxis for mild to moderate hemophilia A cases, exploring a patient-centric approach, situational prophylaxis during high-risk activities, and ...
Results showed a reduction in annualized bleeding rate after a single infusion of fidanacogene elaparvovec compared with standard of care. The Food and Drug Administration (FDA) has approved Beqvez ™ ...
- 94% of patients (51 of 54) remained free from the burden of continuous prophylaxis treatment through five years following a single infusion of HEMGENIX, demonstrating sustained therapeutic benefit - ...
After quite the regulatory process, with Roctavian (valoctocogene roxaparvovec) BioMarin has a potential blockbuster. On June 29th, Roctavian became the first gene therapy approved in the U.S. in the ...
Please provide your email address to receive an email when new articles are posted on . Once-monthly fitusiran prophylaxis improved outcomes compared with on-demand treatment for patients with severe ...
Prophylactic treatment with trimethoprim-sulfamethoxazole (TMP-SMX) significantly reduced the risk for serious infections by approximately 50% in patients with antineutrophil cytoplasmic antibody ...
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