GM1 Gangliosidosis Disease

Learn more about the three onset levels of GM1 disease.

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.

  • Infantile
    Symptoms typically appear between the ages of 3–6 months of age.

  • Juvenile
    Symptoms typically appear between the ages of 2–5 years of age.

  • Late Onset
    Symptoms typically appear in adolescent and into adulthood.

What is GM1 Gangliosidosis Disease?

GM1 gangliosidosis disease (GM1) is a rare lysosomal storage disease 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 baseline of symptoms for as long as possible.

We’re Here to Help You

Getting a diagnosis can be overwhelming. It’s hard to know what to do first. Our Director of Family Support, Becky Benson is here to help. They’ll answer your questions, share information and invite you to connect with our caring and helpful Community.

Research

Learn about ongoing studies, clinical trials, funding opportunities, and research initiatives, and browse our research library.

Family Support

Connect with our Director of Family Support, Becky Benson who will answer your questions and share resources for your specific situation.

We Care for Rare

Learn more about Rare Families and their stories through our We Care for Rare section.