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BridgeBio Pharma is a biotechnology company focused on discovering, developing, testing, and delivering transformative treatments for patients with genetic diseases. The company has four programs in its late-stage pipeline focusing on Mendelian disorders, oncology, and gene therapy. One of its key programs, Attruby (acoramidis) , is an orally administered small molecule designed to stabilize tetrameric transthyretin for the treatment of transthyretin amyloid cardiomyopathy.
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.