There are many diseases with approved drugs developed by a knowledge of genetics such as: myelofibrosis, non-small cell lung cancer (NSCLC), late stage melanoma, chronic myelogenous leukemia (CML), Gaucher's disease, Pompe's disease, hyperphenylalaninemia, Hunter syndrome, mucopolysaccharidosis (MPS) VI, MPS I, Fabry's disease, Type I tyrosinemia, hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency, cystic fibrosis, hereditary angioedema (HAE), cryopryin-associated periodic syndromes, and paroxysmal nocturnal hemoglobinuria3. The gene discussed is NAGS; the disease is mucopolysaccharidosis type 2.