Ultragenyx Pharmaceutical is a biopharmaceutical company focused on identifying, acquiring, developing, and commercializing novel products for treating rare and ultra-rare genetic diseases across Nort... Ultragenyx Pharmaceutical is a biopharmaceutical company focused on identifying, acquiring, developing, and commercializing novel products for treating rare and ultra-rare genetic diseases across North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific region. The company's approved commercial products include Crysvita (burosumab), an antibody for X-linked hypophosphatemia; Mepsevii, an enzyme replacement therapy for mucopolysaccharidosis VII; Dojolvi for long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for homozygous familial hypercholesterolemia. Ultragenyx maintains a robust clinical pipeline featuring multiple late-stage candidates in Phase 3 development, including UX111, an AAV9 gene therapy for Sanfilippo syndrome type A; DTX401, an AAV8 gene therapy for glycogen storage disease type Ia; UX143, a monoclonal antibody for osteogenesis imperfecta; and GTX-102, an antisense oligonucleotide for Angelman syndrome. Founded in 2010 and headquartered in Novato, California, the company operates globally to address unmet medical needs in rare genetic disease treatment.Show lessShow more

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