There is currently no treatment for GM2 gangliosidoses.

phase I/II trials of LYS-GM101 for GM1 gangliosidosis;

Gangliosidosis GM2 where the cat presents with a deficit of a particular enzyme that causes brain damage (can also be screened)

The second candidate medication, LYS-GM101, which targets GM1 gangliosidosis, is expected to enter clinical development in 2019.

Type 1 is the most frequent form of GM1 gangliosidosis but the exact prevalence is not known.

GM1 and GM2 gangliosidosis occur when cats lack particular enzymes that are necessary for proper functioning of the nervous system.

An association between Mongolian spots and inborn errors of metabolism, especially GM1-gangliosidosis and mucopolysaccharidoses, has been described [3].

This is a clinical trial evaluating a planned total of 30 infant or juvenile individuals with a documented gangliosidosis disease.

This is a clinical trial evaluating a planned total of 30 infant or juvenile individuals with a documented gangliosidosis disease.

The gangliosidosis family of disorders (Tay-Sachs, Sandhoff, late-onset Tay-Sachs and GM1 gangliosidosis diseases) are fatal genetic conditions with no known treatment.

The second candidate medication, LYS-GM101, which targets GM1 gangliosidosis, is expected to enter clinical development in 2019.

GM2 gangliosidosis associated with a HEXA missense mutation in Japanese Chin dogs: a potential model for Tay Sachs disease.

Forty-two participants are infants or juveniles who have a gangliosidosis disease; 10 are adults with the late-onset form of disease.

IND (Investigational New Drug) clearance, prerequisite for starting the first-in-human clinical trial of LYS-GM101 in GM1 gangliosidosis

GM1 gangliosidosis is due to an inherited deficiency of the enzyme beta-galactosidase, whereas GM2 gangliosidosis is caused by a lack of the enzyme beta-hexosaminidase.