Ultragenyx Pharmaceutical is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The company's product categories are: biologics, which include Crysvita? (burosumab) for the treatment of X-linked hypophosphatemia and Mepsevii? (vestronidase alfa) for the treatment of Mucopolysaccharidosis VII; small molecules, which includes UX007 for the treatment of long-chain fatty acid oxidation disorders; and gene therapy, which includes DTX301 for the treatment of patients with ornithine transcarbamylase deficiency and DTX401 for the treatment of patients with glycogen storage disease type Ia.
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