Can Gene Therapy Transform the Lives of Patients with GSD1a?

TARGET AUDIENCE
The educational design of this activity addresses the needs of geneticists, hepatologists, endocrinologists, internal medicine physicians, pediatricians, primary care physicians, NPs/PAs, nurses, and other HCPs involved in the care of patients living with GSD1a.

STATEMENT OF NEED/PROGRAM OVERVIEW
For the rare genetic metabolic disorder, glycogen storage disease type 1a (GSD1a), liver transplantation is the only potential cure. Patients endure enormous physical and psychological challenges that significantly impact quality of life. New gene therapies in clinical trials have the potential to replace defective/missing enzymes via adenovirus vector serotype 8 (AAV8) and provide a much-needed treatment option for patients. Clinicians should understand these emerging therapies so they could be prepared to help patients with GSD1a take advantage of these options, when available.

You are invited to join us for an educational activity designed to review the efficacy and safety data on new therapeutic strategies for GSD1a, as well as the rationale for gene therapy and its ability to transcend the limitations of the standard-of-care. Led by a clinical geneticist and a hepatologist, “Can Gene Therapy Transform the Lives of Patients with GSD1a?”, will discuss the diagnosis and differentiation of GSD1a from other GSD types, review the limitations of dietary regulation and transplantation, and describe the mechanisms of action, benefits, and risks associated with emerging gene therapies. This activity will also help clinicians anticipate and mitigate treatment-related adverse events.

EDUCATIONAL OBJECTIVES
After completing this activity, the participant should be better able to:
• Describe the pathophysiology and long-term complications of GSD1a
• Discuss the diagnostic criteria for GSD1a and differentiation from other GSD types
• Recognize the potential of gene therapy to address limitations of the current standard-of-care for GSD1a
• Evaluate clinical data on emerging gene-therapy approaches to GSD1a management