Ultragenyx Pharmaceutical Inc.

Belkins
Ultragenyx is a clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. Our company is led by an experienced management team in rare disease therapeutics. Recognizing that our primary responsibility is to our patients, we are working with advocacy groups to provide support and outreach to individuals and families affected by these disorders and engage them in the clinical testing process. We are also working with regulatory agencies to design and conduct high quality clinical studies that meet the requirements for approval. We are creating an improved model for successful rare disease drug development that increases efficiency and effectiveness by changing the way the process is organized and conducted. We believe that we can deliver significant value to our patients by building a diverse and high-quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

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