The Three Onset Levels
of GM1 Gangliosidosis Disease
GM1 exists on a spectrum with three onset levels. When diagnosing the disease, healthcare providers will determine the onset level based on the age of the affected individual when symptoms first appear.
What is GM1 Gangliosidosis Disease?
GM1 gangliosidosis disease (GM1) is a rare lysosomal storage diseaseLysosomal storage disease is a group of disorders that affect specific enzymes. These enzymes normally break down items for reuse in the cells. If the enzymes are missing or don't work properly the items can build up and become toxic. More where the reduced or absent activity of a vital enzymee called beta-galactosidase (GLB1) causes fatty substances to build up in the nerve cells, which damages the brain and spinal cord.
There is currently no cure for GM1, but there are treatments and therapies to manage symptoms and maintain a baselineBaseline is a technical term for “the new normal” or how things looks when they are stable. With Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases, the baseline shifts, and often it shifts down over time like steps, where each lower step represents a lower level of functioning. More of symptoms for as long as possible.
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