This session will go in depth about some of the gene therapy approvals in 2022 including Skysona (for CALD), Zynteglo (for beta thalassemia), and Hemgenix (for hemophilia B). Attendees will hear from doctors and researchers about the pathway to these therapies, and the outlook on access for those within the various patient populations.
Agenda
Moderator: Rachel Bailey, PhD, assistant professor, UT Southwestern Medical Center
Introduction
12-12:05 p.m.
Recently Approved Therapies
12:05-12:45 p.m.
Speakers:
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Amy Waldman, MD, medical director of the Leukodystrophy Center and pediatric neurologist at Children’s Hospital of Philadelphia
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Alexis Thompson, MD, MPH, chief of the division of hematology at Children's Hospital of Philadelphia
Q&A
12:45-1 p.m.
Thank You to Our Sponsors
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