Systemic sclerosis (SSc) is a chronic disease where the immune system, which normally protects the body from infections and disease, starts to attack the body’s own cells, causing tissue destruction.

Type 1 Gaucher disease, an inherited autosomal recessive disorder, is the most common disease among all inherited glycosphingolipid storage disorders, affecting approximately 7,000 patients worldwide.

Niemann-Pick type C disease (NP-C) is a rare genetic disorder caused by accumulation of fatty molecules such as cholesterol, due to a defect in the movement of fatty molecules within the cells of the body, including the brain.