Gene Therapy: What Does It Mean for Rare Disease Communities?

Gene therapy has the opportunity to be a long-term treatment for people living with rare diseases. Dr. Eric Crombez, Chief Medical Officer of Gene Therapy and Inborn Errors of Metabolism at Ultragenyx, joined Dr. Jeff Bronstein of UCLA Health and Dr. Cary Harding of Oregon Health & Science University in a Facebook Live discussion to better understand the potential benefit of gene therapy and what it means for people living with Glycogen storage disease type Ia (GSDIa), Ornithine Transcarbamylase (OTC) Deficiency
and Wilson disease.

Learn more about gene therapy at Ultragenyx: https://ultragenyx.co/3FzOeIg

#GeneTherapy #RareDiseases #GSDIa, #OTCDeficiency, #WilsonDisease