Ultragenyx Pharmaceuticals

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan diseases, to market. The company focuses on metabolic and rare diseases that may affect small numbers of patients, but for which the medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these clinically underserved diseases. We select target diseases that have well understood biological mechanisms ...


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