Mucolipidosis type IV: clinical manifestations and natural history

Am J Med Genet. 1991 Dec 1;41(3):313-8. doi: 10.1002/ajmg.1320410310.

Abstract

The clinical manifestations and psychomotor development of five patients with mucolipidosis IV (MLIV) from three Ashkenazi-Jewish families are reported. The presenting symptoms were hypotonia, developmental delay, corneal clouding, and puffy eyelids. Four of the patients had convergent strabismus and none progressed beyond a developmental age of 15 months. One patient died of aspiration at 17 years while the oldest patient entered puberty at 20 years, developed a coarse face at 30 years, and is now 32 years old. Histopathological studies in four patients showed storage changes characteristic of MLIV.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Corneal Diseases / genetics*
  • Corneal Diseases / pathology
  • Female
  • Genes, Recessive / genetics*
  • Humans
  • Infant
  • Jews / genetics*
  • Male
  • Mucolipidoses / genetics*
  • Mucolipidoses / pathology
  • Psychomotor Disorders / genetics*
  • Psychomotor Disorders / pathology