McMaster University Researchers Uncover Potential Treatment for Rare Genetic Disorders
McMaster UniversityIn a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and spinal cord. After years of investigating the diseases’ underlying mechanisms, the research team has identified an existing FDA-approved drug that could significantly improve quality of life for affected patients and their families.