RARE

About RARE

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare 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. The Company’s four approved product candidates include Crysvita (burosumab) is for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO); Mepsevii (vestronidase alfa) is for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome; Dojolvi (triheptanoin) is for the treatment of long-chain fatty acid oxidation disorders or LC-FAOD, and Evkeeza (evinacumab) is for the treatment of homozygous familial hypercholesterolemia (HoFH). The Company's clinical product candidates include DTX401, DTX301, UX143, GTX-102, UX701 and UX053.