Providing new hope to families facing the
debilitating aspects of lysosomal storage diseases.
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Disease Overview:
GM2 gangliosidosis, better known as Tay-Sachs Disease, belongs to a grouping of diseases called
lysosomal storage diseases.  These diseases are primarily genetically transmitted and belong to an
even larger class of diseases called Inborn Errors of Metabolism.  

  • Historical Perspective
  • Research Incentive
  • Incidence and Relative Frequency
  • Classifications of LSD's
  • Clinical Presentation
  • Storage Material
  • Enzyme Catabolism
  • Enzyme Replacement Therapy (ERT)
  • Substrate Reduction Therapy (SRT)
  • Pharmacological Chaperone Therapy
  • Gene therapy     
  • Overview
  • Challenges