In addition, inhibitors that specifically target more distal enzymatic steps in Chol biosynthesis have been discovered, among them “AY9944” (trans-1,4-bis(2-chlorobenzylaminoethyl) cyclohexane dihydrochloride), which inhibits DHCR7—the same enzyme that is genetically abnormal in SLOS [28,29] (Figure 1). Here, DHCR7 is linked to Smith-Lemli-Opitz syndrome.