Table 1.

Compounds at 1 µM that Decrease 7-DHC in Dhcr7-Deficient Neuro2a Cells^g

^gAll compounds assayed at 1 µM in 384 well plate. Compounds affect steps promoted by HMG (HMGCoA reductase), Δ8–7, (Δ8–7 isomerase), DR24 (DHCR24 reductase) or CYP51 (CYP).

^aActivity confirmed in human fibroblasts (control or SLOS).

^bCompound not in NIH Clinical Collection.

^cSee ref 59.

^dLanosterol increase in both deficient Neuro2a and fibroblasts.

^eDecrease of all sterol intermediates.

^fSee ref 60.