10/20/2026
This Thursday at Rotary, Developijg Tteatments for Rare Diseases with Emil Kakkas, MD, PhD.
We all have had experiences with disease in ourselves or our families. While there are so many common diseases like diabetes, infections or cancer, there are nearly 10,000 rare diseases that individually are rare but together affect about 10% of the population.
For 95% of these rare diseases there are no treatment available and yet they are very serious diseases.
In the last few years, so much new science and technology is opening the door to treating the most complex diseases that affect humans.
The question remains if we have the technology, why would we not use it, even if the disease is rare. The problem is not just one of science, but now about how our drug regulation and health system works.
We can all agree, if you get something bad that happens to be rare, should we use our best science to treat you?
Dr. Kakkis is best known for his work developing novel treatments for rare diseases and working on policy issues affecting rare and ultrarare disease treatment. He is Chief Executive Officer, President and Founder of Ultragenyx, a company with four approved therapies for five rare diseases and multiple late-stage clinical programs. Dr. Kakkis has received numerous awards
including the Life Science Leadership Pantheon award from California Life Sciences, a Lifetime Achievement Award from the National MPS Society, the Roscoe O. Brady Award for innovation from the WORLDSymposium and BIO’s Henri Termeer Visionary Leadership award for his transformative work in rare diseases. He was honored with the Lifetime Achievement Award at the Bay Area Biotech Forum, San Francisco Business Times (May 2026) and recognized as one of the Bay Areas Most Admired CEOs by the San Francisco Business Times (May 2024).