ADVANCE 2024: Deep Dive Gene Therapy Considerations

Deep Dive: Gene Therapy Considerations
Moderator Introduction: Adam Shaywitz, MD, PhD
Presentation: Gene therapy and the immune system | Barry Byrne, MD, PhD
Presentation: Honoring The Generalizables - Is the Traditional Drug Development Rubric Appropriate for Gene Therapy Research Participants? | Rafael Escandon, DrPH, PhD, HEC-C
Presentation: A combination therapy targeting both primary and secondary pathological storage in Sanfilippo Disease | Alessandro Fraldi, PhD
Presentation: Community perspectives around treatment with gene therapy and long-term follow up | Cara O'Neill, MD, FAAP
Q&A with all presenters
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