Hemophilia B

A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results showed.

In this video, Allyson Pishko, MD, MSCE, highlighted two studies from ASH Annual Meeting and Exposition that investigated fitusiran (Sanofi) as treatment for both hemophilia A and B.

A once-monthly subcutaneous prophylaxis dose of fitusiran appeared safe and effective for people with hemophilia A or B with inhibitors, according to results of a randomized phase 3 study presented at ASH Annual Meeting and Exposition.

The novel gene therapy BAX 335 did not induce sustained factor IX expression among patients with hemophilia B, according to an interim analysis of a phase 1/phase 2 dose-escalation study published in Blood.

This year’s virtual ASH Annual Meeting and Exposition featured hundreds of abstracts that spotlighted research advances in blood cancer and benign hematology research.

The gene therapy etranacogene dezaparvovec conferred a substantial decrease in bleeding events among patients with moderate or severe hemophilia B, according to preliminary data presented at the virtual ASH Annual Meeting and Exposition.

The FDA approved coagulation factor VIIa (recombinant)-jncw for the treatment and control of bleeding episodes among certain adults and adolescents with hemophilia.

ORLANDO, Fla. — In this video at the ASH Annual Meeting and Exposition, Steven W. Pipe, MD, professor of pediatrics and pathology at the University of Michigan, reviews recent innovations in hemophilia and incorporating gene therapy into practice.

ORLANDO – Despite clinical evidence that hemophilia B presents with less severity on a clinical level than hemophilia A, patients report similar quality of life and those with B have more obesity, according to a presentation at ASH Annual Meeting and Exposition.