Generalized Gangliosidoses

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Generalized gangliosidoses, or GM1 gangliosidosis, is a genetic disorder that results in the progressive destruction of neurons in the brain and spinal cord. The condition is often classified into three major types based on age of onset: Infantile (Type 1), Juvenile (Type 2) and Adult (Type 3). GM1 gangliosidoses causes skeletal abnormalities, seizures, loss of vision and profound intellectual disability. It also leads to an enlarged liver, spleen, gums and heart muscle. Life expectancy in those with GM1 gangliosidoses is usually early childhood.   

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