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Inborn Errors of Metabolism
Lysosomal Storage Diseases
GM2 Gangliosidosis
CNS Therapeutic Opportunities
Gene Therapy
Financial Information
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. |