GM2-gangliosidosis. Clinical and biochemical aspects of four cases

Clin Neurol Neurosurg. 1990;92(2):143-8. doi: 10.1016/0303-8467(90)90090-r.

Abstract

We discuss four cases of GM2-gangliosidosis. In one of them the biochemical diagnostic confirmation was difficult. This case revealed striking discrepancies between the results of different methods of enzyme assay. The hexosaminidase A determination based on pH inactivation is not always reliable; assay with natural substrate may be necessary. However, the results with the newly developed substrate 4-MU-GlcNac-6-SO4 are promising and it seems to be a good alternative to the traditional (pH or heat) inactivation procedures. The deficiency can be shown in leukocytes, plasma and fibroblasts with the 6-sulfated substrate. The carrier state seems better reflected in plasma hexosaminidase A than in leukocyte hexaminidase A levels.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Female
  • Hexosaminidase A
  • Humans
  • Infant
  • Male
  • Sandhoff Disease / diagnosis*
  • Sandhoff Disease / enzymology
  • Sandhoff Disease / physiopathology
  • Tay-Sachs Disease / diagnosis*
  • Tay-Sachs Disease / enzymology
  • Tay-Sachs Disease / physiopathology
  • beta-N-Acetylhexosaminidases / deficiency*

Substances

  • Hexosaminidase A
  • beta-N-Acetylhexosaminidases