Treatment Spotlight: CAR-T and Transplant Therapy with Jacob Appelbaum, MD, PhD

This video provides an overview of CAR-T therapy and transplant therapies for people living with ECD. Dr. Appelbaum will review the biology of bone marrow transplant and efforts to distill the impact of transplant for immune function modulation. By the end of the talk, attendees will understand the process of bone marrow transplant, be informed of risks and potential benefits of this lifesaving treatment, and anticipate some of the innovations and challenges anticipated for engineered designer cell therapies including CAR-T cell therapies. This presentation was part of the ECD Patient & Family Gathering held in Seattle, Washington on April 14, 2024. This meeting was hosted by Fred Hutch Cancer Center in partnership with the ECD Global Alliance.

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