Biochemistry in Tables, Schemes, and Graphs - S.D. Zhamsaranova 2009

Amino Acid Metabolism
Inherited Disorders of the Urea Cycle and Their Main Clinical Manifestations

Disease

Enzyme defect

Inheritance pattern

Clinical manifestations

Metabolites

Treatment

Blood

urine

Hyperammonemia type I

Carbamoyl phosphate synthetase I

Autosomal recessive

Coma and death within 24–48 hours of birth

Gln ↑

Ala ↑

Orotate

Hemodialysis, low-protein diet, benzoate, phenylacetate

Hyperammonemia type II

Ornithine transcarbamylase

X-linked

Hypotonia, reduced protein tolerance

Gln ↑

Ala ↑

Orotate

Low-protein

diet,

phenylacetate,

glutamate,

citrulline

Citrullinemia

Argininosuccinate synthetase

Autosomal recessive

Severe neonatal hyperammonemia; in adults, triggered by high-protein load

Citrulline↑

Citrulline↑

Low-protein diet, Arginine, glutamate

Argininosuccinic aciduria

Argininosuccinate lyase

Autosomal recessive

Hyperammonemia, ataxia, seizures, alopecia

Argininosucci

nate↑

Argininosucci

nate,

Gln,

Ala,

Lys

Low-protein diet, arginine

Hyperargininemia

Arginase

Autosomal recessive

Hyperargininemia

Arg↑

Arg,

Lys

Low-protein

diet



Last update: 06/08/2026

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