This is a video synopsis/summary of an Insights involving Allison Wheeler, MD. Wheeler addresses the nuanced considerations for implementing prophylaxis in patients with mild to moderate hemophilia A, ...

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 ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved fidanacogene elaparvovec-dzkt for treatment of adults with moderate to severe hemophilia B. The ...

Prophylactic treatment with trimethoprim-sulfamethoxazole (TMP-SMX) significantly reduced the risk for serious infections by approximately 50% in patients with antineutrophil cytoplasmic antibody ...

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 ...

The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with ...

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 ...

ORLANDO, Florida ― The aggressive chemotherapy regimen given to children with acute lymphocytic leukemia (ALL) during the induction phase, which aims to push the disease into remission, also depletes ...