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Inborn Errors of Metabolism


Lysosomal Storage Diseases
  • Inborn Errors of Metabolism
  • Incidence and Relative Frequency
  • Classifications of LSDs

GM2 Gangliosidosis
  • Clinical Presentation
  • Storage Material
  • Enzyme Catabolism

CNS Therapeutic Opportunities
  • Enzyme Replacement Therapy (ERT)
  • Substrate Reduction Therapy (SRT)
  • Small molecule chaperone drugs
  • Gene therapy     

Gene Therapy
  • Overview
  • Challenges


Financial Information
Providing new hope to families facing the
debilitating aspects of lysosomal storage diseases.