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

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children? At the ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...

When Domenic Catrine was born, his heel swelled up from his vitamin K shot. His circumcision site wouldn't stop bleeding. At the Children's Hospital of Philadelphia, Catrine was diagnosed as having ...

A single intravenous dose of etranacogene dezaparvovec gene therapy maintained factor IX (FIX) activity at 45.7 IU/dL after 5 years in patients with hemophilia B, eliminating the need for prophylaxis ...

At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...

ORLANDO -- Patients with hemophilia B continued to experience low annualized bleeding rates and stable endogenous factor IX expression 5 years after a single infusion of etranacogene dezaparvovec ...

MARBURG, Germany, Feb. 20, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) today announced that the European Commission has granted conditional marketing authorization (CMA) for ...

~ Historic approval represents the first gene therapy in Europe to treat hemophilia B and provides a new treatment option for patients that reduces the rate of annual bleeds, reduces or eliminates the ...

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy ...

Shortly after taking the reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling hemophilia A gene therapy, Roctavian. A year after opting to shrink the ...

In a 13-year follow-up by St. Jude Children’s Research Hospital and University College London published today in The New England Journal of Medicine, Andrew Davidoff, MD, St. Jude Department of ...