For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.

Emil Kakkis; President, Chief Executive Officer, Director; Ultragenyx Pharmaceutical Inc Erik Harris; Executive Vice President, Chief Commercial Officer; Ultragenyx Pharmaceutical Inc Howard Horn; ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...