PlatformQ + ASGCT: Can Gene Therapy Transform the Lives of Patients with GSD1a?

Watch on demand through April 6, 2024!

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.

During this program, speakers David F. Rodriguez- Buritica, MD, and Henry Lin, MD, 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.

Register for this CME program

Learning Objectives

At the conclusion of this program, participants will be 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

Topics of Interest

• Pathophysiology of GSD1a

• Diagnosis of GSD1a and differentiation from other GSD types

• Burden of disease

• Limitations of current standard-of-care

• Emerging gene therapies for GSD1a

• Adeno-associated virus serotype 8 (AAV8) vector

• Safety and efficacy of AAV8 gene therapy for GSD1a or OTC deficiency

• Benefits and risks of gene therapy

• Mitigation of side effects

Register for this CME program