Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are no approved therapies.
About X-linked Hypophosphatemia (XLH): XLH is a hereditary, progressive, and lifelong skeletal disease. It is an X-linked dominant disorder caused by variants in the PHEX gene. XLH is characterized by chronic hypophosphatemia due to increased fibroblast growth factor (FGF23) activity, resulting in rickets and osteomalacia in children and osteomalacia in adults, the sources of progressive and compounding symptoms of XLH.
Rickets and osteomalacia create a significant burden in the daily lives of children. Unresolved symptoms of XLH continue to progress into adulthood, and new symptoms may appear at any age due to ongoing active disease.
Please see brochure via button below for more information and full references. MRCP-KRN23-XXXX. 02/2021
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