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.
Actelion's data registry ‘SMADAR’ (Study MetA DAta Registry), is designed to help patients, caregivers and physicians find the Actelion clinical trials that may be appropriate for them.