Researchers have identified more than seven thousand rare diseases, but effective treatments are available for fewer than ten percent. Drug development for rare diseases is complex given the challenges of the limited, geographically dispersed patient populations, many of which are children.
In this Parexel podcast episode we explore those challenges with rare disease experts:
- Dr. Lucas Kempf, Vice President, Regulatory Affairs at Parexel, who has former rare disease regulatory experience at the FDA
- Sarah Glass, Chief Development Officer at n-Lorem Foundation, a non-profit organization dedicated to charitably provide medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals
- Stacy Hurt, whose youngest son, Emmet suffers from a genetic chromosome abnormality so rare that his syndrome doesn’t have a name.