Cerebral X-linked adrenoleukodystrophy in a girl with Xq27-Ter deletion.

Hershkovitz, Eli; Narkis, Ginat; Shorer, Zamir; Moser, Ann B; Watkins, Paul A; Moser, Hugo W; Manor, Esther · Ann Neurol · 2002

case_report · Level V

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Abstract

An 8.5-year-old girl with a pathogenic mutation (515insC) of the ATP-binding cassette, subfamily D, member 1 gene (ABCD1) on her maternally derived X chromosome showed clinical, biochemical, and magnetic resonance imaging abnormalities similar to those in affected males. Cytogenetic studies led to the surprise finding of a de novo deletion of Xq27 on the paternally derived X chromosome. A bone marrow transplant had an apparently favorable effect. Cytogenetic studies should be performed in all severely symptomatic X-linked adrenoleukodystrophy heterozygotes.

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