Venglustat for treating type 3 Gaucher disease after 1 therapy


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Venglustat is in clinical development for the treatment of patients with Gaucher disease type 3 (GD3) who have been effectively treated with Enzyme Replacement Therapy (ERT) for at least 3 years. Gaucher Disease (GD) is a genetic disorder caused by an enzyme deficiency where fatty substances build up in areas such as the spleen, liver, and bone marrow.

Interventions: Venglustat
Indications: Gaucher disease
Year: 2023

Venglustat is in clinical development for the treatment of patients with Gaucher disease type 3 (GD3) who have been effectively treated with Enzyme Replacement Therapy (ERT) for at least 3 years. Gaucher Disease (GD) is a genetic disorder caused by an enzyme deficiency where fatty substances build up in areas such as the spleen, liver, and bone marrow. This builds up to cause organ enlargement and can affect their function. GD3 is the chronic neuropathic form of the disease. GD3 affects the central nervous system and causes neurological symptoms including eye movement disorders, seizures, and other cognitive difficulties. There are currently no approved therapies to treat the neurological manifestations of GD3. Patients with GD3 can currently receive ERT or substrate replacement therapy to address symptoms not involving the brain, like organ enlargement and bone issues.