Ultragenyx Pharmaceutical Inc. is a development-stage biopharmaceutical company focused on the identification, acquisition, development, and commercialization of products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. The Company focuses on diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. The Company�s pipeline consists of two product categories: biologics, including a monoclonal antibody and enzyme replacement therapies, and small-molecule substrate replacement therapies. The Company�s product include KRN23 (UX023) for the treatment of XLH, rhGUS (UX003) for the treatment of MPS 7, rhPPCA (UX004) for the treatment of galactosialidosis, Triheptanoin (UX007) for the treatment of LC-FAOD, Triheptanoin (UX007) for the treatment of Glut1 DS and SA-ER (UX001) for the treatment of HIBM.