Abstract submission is now open! Send us your research through Jan. 26, 2024 to present at the 27th Annual Meeting.
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
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
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
May 7-11, 2024 | Baltimore, MD
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