Gene Therapy for Hemophilia B"The Next Step Forward in Disease Management

Target Audience
This educational activity is directed toward hematologists and hematologist-oncologists. Pediatricians, nurses, nurse practitioners, physician assistants, and other clinicians involved in the management of patients with hemophilia B are invited to attend.

Activity Overview
Gene therapy for hemophilia B has the potential to reduce the treatment burden for patients and their care partners by eliminating the need for regular prophylaxis through long-term expression of endogenous factor IX at levels sufficient to provide protection from bleeding. Challenges associated with gene therapy still need to be addressed to ensure widespread therapeutic use, but it offers a promising option for this patient population. In this program, expert faculty will assess clinical trial data for gene therapy in patients with hemophilia B, review strategies regarding patient selection for gene therapy, and discuss the role of the multidisciplinary team in the ongoing management of patients with hemophilia B.

Learning Objectives
Upon completion of this activity, participants should be better able to:

Outline the current standard of care for patients with hemophilia B
Assess clinical trial data for gene therapy for hemophilia B
Apply strategies to select appropriate patients for gene therapy
Review the role of the multidisciplinary team in the management of patients receiving gene therapy for hemophilia B