[Niemann-Pick disease, type A: a case report]

Neurol Neurochir Pol. 2006 May-Jun;40(3):238-42.
[Article in Polish]

Abstract

Niemann-Pick disease (NPD) type A is a rapidly developing metabolic illness, with autosomal recessive mode of inheritance. A deficiency of the lysosomal enzyme--acid sphingomyelinase (ASM) produces the clinical phenotype with multiple organ involvement including the central nervous system. Type A NPD is characterized by failure to thrive, hepatosplenomegaly and rapidly progressive neurodegenerative course that leads to death by the age of 2-3 years. The authors report a 3-year-old boy with fatal course of the disease.

Publication types

  • Case Reports
  • English Abstract

MeSH terms

  • Child, Preschool
  • Fatal Outcome
  • Frontal Lobe / pathology
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Niemann-Pick Diseases / diagnosis*
  • Niemann-Pick Diseases / genetics
  • Parietal Lobe / pathology