PRF co-founder serves as thought leader in rare disease drug development

PRF’s co-founder and Medical Director, Dr. Leslie Gordon, was recently invited to contribute to an educational video series produced by the National Organization for Rare Disorders (NORD), along with her colleague Dr. Francis Collins, Science Advisor to the President of the U.S. and former Director of the National Institutes of Health (NIH).

The two were asked to share learnings from their experiences in Progeria research, starting as far back as the development of PRF’s International Registry in 1999. In the two videos below, they share takeaways on the Progeria gene finding in 2003, through the development of PRF’s medical & research database, PRF’s natural history study, and finally through the Food and Drug Administration (FDA) approval of lonafarnib, the first-ever treatment for Progeria, to demonstrate possible pathways to advancing research on rare diseases for those involved in rare disease research and advocacy.

This full series is deeply informative for anyone interested in drug development in rare disease, and the interviews with Dr. Gordon and Dr. Collins are located within the module on Natural History Studies within this series.