Facebook
Twitter
LinkedIn
Forward| Ultragenyx Pharmaceutical - Exhibitor | |
|---|---|
Thomas Vondra Tasha Bergman |
At Ultragenyx, our goal is to bring novel therapies to patients with serious rare and ultra-rare genetic diseases. We are targeting multiple therapeutic areas in which we have deep expertise: endocrine/bone diseases, metabolic diseases, and central nervous system (CNS)/muscle diseases. Our team has a proven track record of identifying, developing, and launching medicines for rare diseases. Since our founding in 2010, we’ve launched three FDA-approved approved treatments in four indications, and developed a diverse portfolio of investigational therapies focused on modifying diseases. We put patients and their caregivers at the center of our work, involving them at all stages of therapeutic development. This provides us with an understanding of the impact a disease has on a patient – biologically, functionally, and emotionally – so we can address the entire person and not just their symptoms. All of our efforts – from translational research to clinical development to patient access programs – are rooted in our commitment to provide patients and their families with courage and confidence today. Additional Information |
