Mulibrey nanism: three additional patients and a review of 39 patients

Am J Med Genet. 1995 Jan 30;55(3):349-55. doi: 10.1002/ajmg.1320550320.

Abstract

We report on 3 patients with Mulibrey nanism (MN), or Perheentupa syndrome: the first 2 sibs from Argentina and a new patient from Spain. All 3 patients had growth failure, short stature, abnormal pigmentary retinal changes, and a J-shaped sella turcica. These findings are considered major criteria of MN. Two had pericardial constriction, which is a frequent and life-threatening abnormality in this syndrome. MN is a rare autosomal recessive condition. Reviewing the 39 patients described so far, we have classified the anomalies into the very frequent (present in more than 66%), frequent (in at least 25%), and not frequent. Identifying the anomalies specific to MN should help its early diagnosis and treatment.

Publication types

  • Case Reports

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Cerebral Ventricles / abnormalities
  • Child, Preschool
  • Dwarfism / genetics*
  • Eye Abnormalities / genetics
  • Face / abnormalities
  • Genes, Recessive
  • Hepatomegaly / genetics
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Muscle Hypotonia / genetics
  • Pericarditis, Constrictive / genetics*
  • Sella Turcica / abnormalities*
  • Syndrome