Pulmonary arterial hypertension
Pulmonary arterial hypertension is a serious disease of the arteries connecting the lungs to the heart (the pulmonary arteries).
Digital ulcers
Digital ulcers are very painful and result in difficult-to-heal open sores that occur on fingers and toes.
Type 1 Gaucher disease
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
Niemann-Pick type C disease is a rare genetic lysosomal storage disorder that causes severe, progressive neurological symptoms.
