How GM1 Gangliosidosis Presents

GM1 gangliosidosis is a rare, genetically inherited lysosomal storage disease. There are three main GM1 subtypes, but overall GM1 primarily affects children, bringing about life-limiting neurological and developmental debilitations, among others. Read the full article on GM1 gangliosidosis here.

Onset of GM1 gangliosidosis can take place during the first year of life up until a person is in early adulthood, depending on the subtype. In some cases of infantile (Type 1) GM1, the disease can be discovered in utero.

Symptoms of the disease can present in myriad ways with each GM1 subtype. The most extreme and severe symptoms occur with the infantile form of the disease, and the least severe symptoms occur with adult onset. Severity correlates to activity of the beta-galactosidase enzyme. Those with more beta-galactosidase, who have later onset types of the disease, experience subsequently less severe, but no less life-limiting symptoms of the disease.