Ultragenyx Pharmaceutical Inc
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company 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 clinical-stage pipeline consists of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. Its products include Crysvita (burosumab), Mepsevii (vestronidase alfa), Dojolvi (triheptanoin), and Evkeeza (evinacumab). Crysvita is used for the treatment of X-linked hypophosphatemia, or XLH, in adult and pediatric patients one year of age and older. Mepsevii is used for the treatment of children and adults with mucopolysaccharidosis VII, or MPS VII, also known as Sly syndrome. Dojolvi is used for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders, or LC-FAOD. Evkeeza is used for the treatment of homozygous familial hypercholesterolemia, or HoFH.
Settore
Healthcare
Industria
Biotechnology & Drugs
Paese
United States
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