This includes hemophilia A and lipoprotein lipase deficiency, as well as several neurological conditions such as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), aromatic L-amino acid decarboxylase (AADC) deficiency, and Leber congenital amaurosis (LCA). The gene discussed is LPL; the disease is Leber congenital amaurosis.