Leading the future of rare disease medicine for patients like Mason.
We use our experience, insight, and commitment to move the rare disease community forward. Our goal is to provide medicines to those who have no other choices and to help patients face rare diseases head on, with courage and passion.
LEARN MORE ABOUT ULTRAGENYX
At Ultragenyx, our goal is to bring novel therapies to patients with serious rare and ultra-rare genetic diseases who lack approved therapies to treat their underlying disease.