During our conversation with Dr. Stephanie Cherqui at the Invest in Cures event by LaunchBio, we explored the transformative impact of gene therapy in treating genetic disorders, especially rare diseases.
Dr. Cherqui is a professor at the University of California San Diego, Department of Pediatrics. She received her Ph.D in 2002 at Necker hospital (Paris, France), her research project focused on the molecular characterization of cystinosis, a metabolic hereditary disease
The message I give to all students, to all researchers, everyone who works in rare diseases: you have to hand on, do not listen to anyone telling you that because it is rare that it is not worth it. It is worth it for the patients, for their families.
Dr. Stephanie Cherqui
Even though rare diseases affect fewer people, each treatment has a profound impact on patients and their families. The knowledge gained can help treat many other conditions in the future. Dive into her inspiring thoughts on the importance of perseverance and collaboration in rare disease research.
