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Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
Date:8/15/2011

ients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for HIBM.

UX001 is an extended release formulation of sialic acid that should allow the maintenance of steady levels of sialic acid after oral administration. The sialic acid replacement therapy is expected to restore the proper biochemistry of glycoproteins and glycolipids by allowing proper sialylation.  Data from an HIBM mouse model show a profound beneficial effect of sialic acid replacement therapy on the biochemistry, pathology and clinical outcomes of HIBM mice.

About Ultragenyx

Ultragenyx is a private, clinical-stage biotechnology company committed to developing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases. Ultragenyx is leveraging its experience in orphan indications to identify target diseases with well understood biological mechanisms to generate a pipeline of promising treatments for medically underserved patient populations. Ultragenyx' lead program, UX001, is currently being evaluated in a Phase 1 trial in hereditary inclusion body myopathy (HIBM).

Ultragenyx aspires to create a new model for successful rare disease drug development and to deliver significant value to all its key stakeholders by rapidly and efficiently transforming good science into great medicines for untreated rare diseases. Ultragenyx' investors include TPG Biotech and Fidelity Biosciences, HealthCap and Pappas Ventures. The company is led by Emil Kakkis, MD, PhD, a recognized industry leader in rare disease research and development.

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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