This panel discussion about recent Rare Breakthroughs in Research and Care was held at the 2024 NORD Living Rare, Living Stronger Patient and Family Forum on June 8, 2024 in Los Angeles, California.
It covers major updates from doctors in the field alongside videos of their patients explaining the successful treatments they underwent for various rare diseases, including severe combined immunodeficiency (SCID) and childhood leukemia. Innovations discussed include stem cell transplants, precision medicine, immunotherapy, CAR T-cell therapy, genome sequencing of tumors, and more.
Panelists included Dr. Donald B. Kohn of UCLA and the California Institute for Regenerative Medicine (CIRM); Dr. Amanda Marinoff, pediatric oncologist at UCSF Benioff Children's Hospitals, a NORD Rare Disease Center of Excellence; and Dr. Sheldon Shuster, President of the Keck Graduate Institute.
Special thanks to the families who shared their stories as part of this session.
Learn more at livingrare.org.
15 окт 2024
