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Treatment of Lysosomal Storage Diseases
A repurposed FDA-approved drug for effective treatment of Sandhoff disease
Technology Overview
Researchers at McMaster University have discovered that a repurposed FDA-approved drug is an effective treatment against Sandhoff disease in mice models. Treated mice had an increased life span and reduced muscle atrophy compared to control.
Benefits
- Targets a unique pathway compared to other therapeutic methods
- Can be paired with additional substrate reduction drugs or enzyme replacement treatments
Applications
- Treating Tay Sachs and Sandhoff disease
Opportunity
McMaster University is seeking for technology licensing opportunities and research collaboration.
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