CSF3 and Niemann-Pick disease, type C1: Although patient populations were small, it is of interest that the two patients with the highest plasma G-CSF concentration were the ones that eventually received an intervention, either being miglustat, a sphingolipid synthesis inhibitor that has been approved as the first drug specifically targeted for NPC1 disease in 2009 (in Europe), and delays disease progression and improves neurological symptoms (50, 51), or a liver transplantation.