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.
  

Inborn Errors of Metabolism

• Historical Perspective
• Research Incentive

   

Lysosomal Storage Diseases

• Incidence and Relative Frequency
• Classifications of LSD’s

GM2 Gangliosidosis

• Clinical Presentation
• Storage Material
• Enzyme Catabolism

CNS Therapeutic Opportunities

• Enzyme Replacement Therapy (ERT)
• Substrate Reduction Therapy (SRT)
• Pharmacological Chaperone Therapy
• Gene therapy     

Gene Therapy

• Overview
• Challenges