Bio

I have spent my career so far seeking to develop new treatments for rare diseases that built upon my training in biochemistry, medicine and research. I have had the good fortune to succeed in developing and getting FDA approval for 3 medicines for rare disease patients, and getting another 3 into clinical studies. My goals going forward are to work on improving the regulatory process that controls how rare disease treatments get developed. I have gone back to biotech again in 2010, starting Ultragenyx Pharmaceutical Inc. We now will have 4 products in 5 clinical programs this year and team of about 70 people. We have gone public in an IPO in January 2014. The development team are working to treat some of the many untreated rare diseases and focused on translating these therapies as promptly as possible. It can never be fast enough. Specialties: Drug development in rare diseases Biochemical genetic disease treatment Mucopolysaccharidoses and their clinical management