Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a serious disease of the arteries connecting the lungs to the heart (the pulmonary arteries).
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 is a rare genetic lysosomal storage disorder that causes severe, progressive neurological symptoms.
Dupuytren's disease is a common and disabling fibroproliferative condition of the hand, which may progress slowly over time to Dupuytren's contracture.
Peyronie’s (pronounced " pay-ROH-neez ") disease is the development of scar tissue, also known as a Peyronie’s plaque, under the skin of the penis, which causes it to bend or curve during an erection.