Abstract
We report the clinical, biochemical and molecular findings on the first documented patient with 4-hydroxybutyric aciduria (4-HBA, McKusick 271980) from Uruguay. The patient displayed a severe picture and turned out to be homozygous for a mutation (c.1226G < A) previously shown to be associated with null enzyme activity.
MeSH terms
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Amino Acid Metabolism, Inborn Errors / diagnosis
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Amino Acid Metabolism, Inborn Errors / genetics*
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Amino Acid Metabolism, Inborn Errors / metabolism
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Homozygote
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Humans
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Hydroxybutyrates / urine*
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Infant
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Male
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Mutation, Missense
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Succinate-Semialdehyde Dehydrogenase / deficiency*
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Succinate-Semialdehyde Dehydrogenase / genetics*
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Uruguay
Substances
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Hydroxybutyrates
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4-hydroxybutyric acid
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Succinate-Semialdehyde Dehydrogenase