Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The Company's therapies and clinical-stage pipeline consist of four product categories, such as biologics, small molecules, gene therapy and nucleic acid product candidates. Its three approved product candidates include Crysvita (burosumab), for the treatment of X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa), for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome and Dojolvi (triheptanoin), for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). Its clinical product candidates include DTX401, for the treatment of glycogen storage disease type Ia (GSDIa), DTX301, for the treatment of ornithine transcarbamylase (OTC), UX143, GTX-102, UX701 and UX053.