Venglustat is currently in clinical development for the treatment of adult and paediatric patients with GM2 gangliosidosis (which includes Tay-Sachs disease and its more severe form; Sandhoff disease) as well as other rare disorders including GM1 gangliosidosis, galactosialidosis and sialidosis. All these conditions are caused by genetic mutations involving a mechanism called the glucosylceramide-based sphingolipid pathway that results in an inability to properly break down large fatty molecules called lipids. The resulting abnormal build-up of lipids can cause damage in the cells and tissues in the brain and nervous system. Symptoms vary between the individual conditions but can include motor and muscle weakness, speech and swallowing problems, skeletal complications, seizures and loss of vision. There are currently no approved treatment options for GM2 and GM1 gangliosidosis, galactosialidosis and sialidosis.
Venglustat is an investigational therapy small-molecule drug that
Venglustat for treating late onset GM2 gangliosidosis, GM1 gangliosidosis and other ultra-rare disease
Venglustat is currently in clinical development for the treatment of adult and paediatric patients with GM2 gangliosidosis (which includes Tay-Sachs disease and its more severe form; Sandhoff disease) as well as other rare disorders including GM1 gangliosidosis, galactosialidosis and sialidosis. All these conditions are caused by genetic mutations involving a mechanism called the glucosylceramide-based sphingolipid pathway that results in an inability to properly break down large fatty molecules called lipids.
Interventions:
Venglustat
Year:
2023