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February 23, 2023
Speakers focused on gene therapy approvals in 2022 including Skysona (for CALD), Zynteglo (for beta thalassemia), and Hemgenix (for hemophilia B).
Moderator: Rachel Bailey, PhD, assistant professor, UT Southwestern Medical Center
Introduction
Recently Approved Therapies
Speakers:
Amy Waldman, MD, medical director of the Leukodystrophy Center and pediatric neurologist at Children’s Hospital of Philadelphia
Alexis Thompson, MD, MPH, chief of the division of hematology at Children's Hospital of Philadelphia
Q&A
September 23-24, 2024 | Washington, D.C.
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