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Hyperion Therapeutics Announces Enrollment of First Patient in Phase 1/2 Clinical Trial of GT4P in Patients With Urea Cycle Disorders
Date:10/23/2007

d synthetase (AS)), and a pre-prodrug of phenylacetate (PAA).

About Urea Cycle Disorder

A urea cycle disorder is an inherited, inborn error of metabolism present in an estimated 1 in 10,000 live births in the U.S. Patients with UCDs lack or are deficient in one of the key enzymes that comprise the urea cycle, the body's primary vehicle for removing ammonia, a potent neurotoxin, from the bloodstream. The age of onset of UCDs is typically in the neonatal period, but onset can occur at any age, depending on the severity of the disorder. Left untreated, UCDs can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.

There is no cure for UCDs. Adherence to a low-protein diet is a key component to chronic disease management. Current therapies include arginine supplementation, which drives functional parts of the urea cycle, essential amino acid supplementation, replacement of urea cycle intermediates, and administration of nitrogen-scavenging drugs including BUPHENYL(R) for chronic management and AMMONUL(R) (sodium phenylacetate and sodium benzoate) disease Injection 10%/10% for episodes of acute hyperammonemia. Orthotopic liver transplantation may also be considered for patients with severe disease.

About Hyperion Therapeutics

Hyperion Therapeutics is a specialty therapeutics company focused on becoming a global leader in gastrointestinal (GI) and hepatology therapeutic programs and products that address underserved patient populations or unmet medical needs. The company has assembled a seasoned executive team with extensive industry experience developing and commercializing specialty pharmaceutical products. Hyperion is headquartered in South San Francisco, CA. For additional information, visit: http://www.hyperiontx.com.

About AMMONUL(R)

AMMONUL(R) is indicated as adjunctive therapy for the treatment
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SOURCE Hyperion Therapeutics, Inc.
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