SI
Ultragenyx Pharmaceutical Pipeline
| Drug | Indication | Phase |
|---|---|---|
| Crysvita® (burosumab) | X-linked Hypophosphatemia (XLH) | Approved/Commercial |
| Dojolvi® (triheptanoin) | Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) | Approved/Commercial |
| Mepsevii® (vestronidase alfa) | Mucopolysaccharidosis VII (MPS VII) | Approved/Commercial |
| DTX401 | Glycogen Storage Disease Type Ia (GSDIa) | Phase 3 |
| UX701 | Wilson Disease | Phase 1/2/3 |
| UX143 (setrusumab) | Osteogenesis Imperfecta (OI) | Phase 3 |
| GTX-102 | Angelman Syndrome | Phase 1/2 |
| UX053 | Glycogen Storage Disease Type III (GSDIII) | Phase 1 |
Leadership Team at Ultragenyx Pharmaceutical
ED
Emil D. Kakkis
Founder, Chief Executive Officer and President
SS
Sharon Shacham
Chief Scientific Officer
EC
Eric Crombez
Chief Medical Officer
TH
Ted Huizenga
Chief Regulatory Officer
KP
Karah Parschauer
Chief Legal Officer and Corporate Secretary
MR
Matthew R. Patterson
Chairman of the Board
DK
Dennis Karl
Chief Operating Officer
HH
Howard Horn
Chief Financial Officer
CL
Camille L. Bedrosian
Chief Medical Officer, Rare Genetic Diseases
MC
Mardi C. Dier
Executive Vice President, Chief Financial Officer