GSD1a From Today to Tomorrow: Navigating Today's Challenges & Looking Ahead into the Gene Therapy Pipeline (This activity is supported by Ultragenyx)
19 Mar 2025
Venue:
JW Marriott Los Angeles LA LIVE
Meeting Room:
Gold Ballroom 4
ACMG offers industry and related organizations the opportunity to host non-commercial, grant-supported educational satellite sessions in conjunction with the meeting, enhancing the educational experience for attendees. Corporate Educational Satellite Sessions are planned solely by corporations/industry, ACCME providers, medical education companies and/or non-profit organizations. These sessions focus on highlighting new developments, generating interest in innovative products or services, and sharing the latest insights with meeting participants. These activities are not part of the official ACMG meeting programming and are not accredited by ACMG.
Symposium to feature 3 expert speakers and moderated panel discussion including Q&A.
Faculty
Dr. Areeg El-Gharbawy, Duke University Medical Center
Dr. Carlos Prada, Lurie Children's Hospital, Northwestern University
Dr. Steven Pipe, C.S.Mott Children's Hospital, University of Michigan
Pre-registration is required.
For more information and to register
Symposium to feature 3 expert speakers and moderated panel discussion including Q&A.
Faculty
Dr. Areeg El-Gharbawy, Duke University Medical Center
Dr. Carlos Prada, Lurie Children's Hospital, Northwestern University
Dr. Steven Pipe, C.S.Mott Children's Hospital, University of Michigan
Pre-registration is required.
For more information and to register
Learning Objectives
- Review unmet needs and challenges that persist in GSD Ia despite SOC
- Review gene therapy approaches under development for GSD Ia (focus on the approaches/technology e.g. gene transfer, gene editing, mRNA, etc)
- Gene therapy: Practical considerations for Implementation at clinical centers
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