GM1 Gangliosidosis Type 3 – Adult
Type 3 is the rarest form of GM1 gangliosidosis. The age of onset for Adult GM1 has been reported to vary widely and can occur anywhere from age 3 to 30. Typically onset of notable symptoms occurs sometime in adulthood. Life expectancy can extend well into adult life.
In this chronic, adult form of the disease, the symptoms may be more mild compared to Types 1 and 2. There is also often extreme clinical variability between patients. In adult GM1, there is an inverse correlation between disease severity and residual enzyme activity.
The adult-onset form of GM1 is more frequent in the Japanese population.
Symptoms of GM1 Gangliosidosis Type 3
- Dystonia
- Speech disturbance
- Imbalance; gait disorders
- Ataxia
- Uncoordinated movements
- Speech problems
- Visual problems
- Vertigo
- Slowly progressive dementia
- Intellectual degradation
- Skeletal dysplasias that can include spine deformities
- Skeletal muscle atrophy
- Most reported cases are in patients of Japanese origin
Learn about Lindsay’s life after being diagnosed with adult GM1 gangliosidosis.
Read Their Stories
Lindsay’s experience
“She is aware of how GM1 is taking over her body and she can do fewer and fewer things, she still walks but it is agony to think how much time is left, how much time is left to listen to that thread of voice, or how much time is left to use your hands.”
María José, mother of a 20-year-old daughter living with Type 3, adult onset GM1
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