RARE

Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. The company identifies, acquires, develops, and commercializes novel products for the treatment of serious, rare, and ultra-rare diseases, with a focus on debilitating genetic conditions. Its medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients aged one year and older. Mepsevii is indicated for pediatric and adult patients for the treatment of Mucopolysaccharidosis VII. The company operates across North America, Latin America, Europe, the Middle East and Africa, and Asia-Pacific, with the majority of its revenue generated from North America.