X-linked lissencephaly with abnormal genitalia associated with renal phosphate wasting

Neuropediatrics. 2004 Jun;35(3):202-5. doi: 10.1055/s-2004-817955.

Abstract

X-linked lissencephaly with abnormal genitalia (XLAG) is a rare disorder caused by mutations in the aristaless-related homeobox (ARX) gene. We report on the clinical data of a boy with a 1-bp deletion (790 delC) resulting in a frame shift in the ARX gene and prolonged survival until age 18 months. Similar to other patients, the boy showed postnatal microcephaly, hypothalamic dysfunction, intractable neonatal seizures, and chronic diarrhoea. In addition, he suffered from exocrine pancreatic insufficiency and renal phosphate wasting became apparent from age 5 months, both of which have not been described previously in XLAG. This allows us to speculate that the phenotype of XLAG is more complex than hitherto known and may include renal phosphate wasting which might not have been observed in other patients due to early death.

Publication types

  • Case Reports

MeSH terms

  • Abnormalities, Multiple / diagnosis*
  • Abnormalities, Multiple / genetics
  • Cerebral Cortex / abnormalities*
  • Exocrine Pancreatic Insufficiency / diagnosis
  • Exocrine Pancreatic Insufficiency / genetics
  • Genes, Homeobox / genetics
  • Genetic Diseases, X-Linked / diagnosis*
  • Genetic Diseases, X-Linked / genetics
  • Homeodomain Proteins / genetics
  • Humans
  • Infant, Newborn
  • Kidney / metabolism
  • Male
  • Phenotype
  • Phosphates / metabolism
  • Transcription Factors / genetics
  • Urogenital Abnormalities / diagnosis*
  • Urogenital Abnormalities / genetics
  • Wasting Syndrome / diagnosis*
  • Wasting Syndrome / genetics

Substances

  • ARX protein, human
  • Homeodomain Proteins
  • Phosphates
  • Transcription Factors