A new case of alpha-N-acetylgalactosaminidase deficiency with angiokeratoma corporis diffusum, with Ménière's syndrome and without mental retardation

Br J Dermatol. 2001 Feb;144(2):363-8. doi: 10.1046/j.1365-2133.2001.04028.x.

Abstract

alpha-N-acetylgalactosaminidase (alpha-NAGA) deficiency is a rare hereditary lysosomal storage disease, and only three alpha-NAGA-deficient patients with angiokeratoma corporis diffusum (Kanzaki) have been described. We report a further case in a 47-year-old Japanese woman, the product of a consanguineous marriage. The remarkable findings in this patient were her normal intelligence, Ménière's syndrome, disturbance of peripheral sensory nerves, hearing loss and cardiac hypertrophy. alpha-NAGA enzyme activity in her plasma was 0.77% of the normal value. Other enzyme activities, such as alpha-galactosidase, beta-galactosidase, alpha-L-fucosidase, beta-mannosidase and aspartylglucosaminidase, were within normal limits. A large quantity of amino acid O-glycans was detected in her urine. Gene analysis revealed a novel point mutation (G-->A transition) at nucleotide 11018 (986 in the cDNA) resulting in an Arg-329-Gln substitution. Kanzaki disease has the same enzyme defect as Schindler disease, but the manifestations are quite different.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Fabry Disease / complications*
  • Fabry Disease / pathology
  • Female
  • Hexosaminidases / deficiency*
  • Humans
  • Intellectual Disability
  • Lysosomal Storage Diseases, Nervous System / complications
  • Lysosomes / ultrastructure
  • Meniere Disease / etiology*
  • Middle Aged
  • alpha-N-Acetylgalactosaminidase

Substances

  • Hexosaminidases
  • NAGA protein, human
  • alpha-N-Acetylgalactosaminidase