Abstract
Children with symptoms of profound biotinidase deficiency with null mutations are more likely to have hearing loss develop than those with missense mutations, even if not treated for a period of time. Hearing loss appears to be preventable in children with null mutations if treatment is initiated soon after birth.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Adolescent
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Age of Onset
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Biotinidase Deficiency / diagnosis
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Biotinidase Deficiency / genetics*
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Biotinidase Deficiency / therapy
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Child, Preschool
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Consanguinity
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Female
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Frameshift Mutation / genetics
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Genotype
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Hearing Loss / diagnosis
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Hearing Loss / genetics*
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Humans
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Infant
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Infant, Newborn
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Male
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Mutation, Missense / genetics
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Phenotype