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Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
Date:10/5/2011

 Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for GNE Myopathy, also known as hereditary inclusion body myopathy (HIBM).

The company is led by Emil Kakkis, MD, PhD, a recognized industry leader in rare disease research and development, and he has assembled an experienced management team in rare disease therapeutics.  Ultragenyx is creating an improved model for successful rare disease drug development that has the potential to increase efficiency and effectiveness by changing the way the process is organized and conducted. The company believes that it can deliver significant value to patients by building a diverse and high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.


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SOURCE Ultragenyx Pharmaceutical Inc.
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