Rare diseases
Investigational molecules
Locations
60 Leveroni Court Suite 200, 94949-5746, United States
Ultragenyx Pharmaceutical Inc. funds clinical research in glycogen storage disease type Ia, ornithine transcarbamylase deficiency, and long-chain fatty acid oxidation disorders, with a strong focus on restoring metabolic control and reducing disease burden in inherited conditions affecting energy and ammonia handling.
Its research portfolio also includes Wilson disease, reflecting interest in disorders of copper regulation and broader inborn errors of metabolism.
Ultragenyx Pharmaceutical Inc. runs clinical trials in conditions such as Glycogen storage disease type I, Angelman's syndrome, Mucopolysaccharidosis III, Ornithine transcarbamoylase deficiency, among others.
Clinical trials run by this sponsor – those enrolling participants first.
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