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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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