Researchers complete final analysis of a phase 3 study of men with hemophilia B receiving intravenous etranacogene dezaparvovec.

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, ...

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced positive topline results from the Phase 3 AFFINE study (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene ...

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 ...

New FRONTIER5 data show that a direct switch to investigational Mim8 (denecimig) prophylaxis treatment from emicizumab, without the need for a washout period, was well-tolerated with no safety ...

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults ...

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 ...

Prophylactic cardiac treatment in DMD patients is associated with a 54% lower hazard of death, highlighting its potential survival benefit. Only 27.7% of DMD patients received prophylactic cardiac ...

An antisense oligonucleotide therapy showed efficacy as a prophylactic treatment for hereditary angioedema (HAE), the phase III OASIS-HAE trial showed. The least-squares mean time-normalized HAE ...