Diagnostic and treatment challenges of neuronopathic Gaucher disease: two cases with an intermediate phenotype

J Inherit Metab Dis. 2004;27(5):687-90. doi: 10.1023/b:boli.0000043027.80328.75.

Abstract

Gaucher disease (GD) is a lysosomal storage disorder with a broad, overlapping clinical spectrum. The presented two case reports highlight the clinical evaluation required in neuronopathic GD to assist with medical management and genetic counselling.

Publication types

  • Case Reports

MeSH terms

  • Age of Onset
  • Child, Preschool
  • Disease Progression
  • Female
  • Fluoroscopy
  • Gaucher Disease / diagnosis*
  • Gaucher Disease / therapy*
  • Genes, Recessive
  • Humans
  • Male
  • Phenotype
  • Prognosis