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Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
Scholar Rock Holding Corp is a late-stage biopharmaceutical company focused on discovering, developing, and delivering innovative medicines for treating serious diseases in which signaling by protein growth factors plays a fundamental role. Its pipeline includes Neuromuscular and Obesity, Immuno-oncology, Fibrotic Disease, and Hematology. The group operates and manages its business as a single segment to assess performance and make operating decisions.