JM

John M. Higgins

Chief Business Officer

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Pipeline

DrugIndicationPhase
DTX301Ornithine Transcarbamylase (OTC) DeficiencyPhase 3
UX053Glycogen Storage Disease Type III (GSDIII)Phase 3
UX016GNE MyopathyPhase 1/2
DTX401Glycogen Storage Disease Type Ia (GSDIa)Phase 3
GTX-102Angelman SyndromePhase 1/2
UX701Wilson DiseasePhase 1/2
Crysvita (burosumab)X-Linked Hypophosphatemia (XLH)Approved
Dojolvi (triheptanoin)Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)Approved