In 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. "Sandhoff and Tay-Sachs are devastating diseases," says Suleiman Igdoura, a professor of biology and pathology who has been researching these conditions for years.
"They're marked by progressive loss of motor functions - from sitting, standing, and swallowing to even breathing - as neurons in the nervous system die. Watching someone go through this is heartbreaking." Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often proving fatal within a few years.
In rare cases, symptoms of Tay-Sachs and Sandhoff disease appear later in childhood or even in early adulthood, progressing more slowly and offering a longer—but still profoundly challenging—life course. Patients often require intensive hospital care as symptoms worsen, and our current treatment options are severely limited. But now, there's hope.
" Suleiman Igdoura, professor of biology and pathology By studying late-onset cases, Igdoura and his team uncovered that t.
