Lunch and Learn: Recently Approved Therapies

February 23, 2023

Speakers focused on gene therapy approvals in 2022 including Skysona (for CALD), Zynteglo (for beta thalassemia), and Hemgenix (for hemophilia B).

Agenda

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