simvastatin + cholesterol

This combination therapy consists of simvastatin, an HMG-CoA reductase inhibitor, and dietary cholesterol supplementation, specifically formulated in an OraPlus oral suspending vehicle. It was developed for the treatment of Smith-Lemli-Opitz syndrome (SLOS), a metabolic disorder characterized by a deficiency in the enzyme 7-dehydrocholesterol reductase (DHCR7), which leads to low systemic cholesterol and the accumulation of toxic 7-dehydrocholesterol (7-DHC). Simvastatin is hypothesized to upregulate the expression of the defective DHCR7 enzyme or alternative metabolic pathways to increase endogenous cholesterol production and lower 7-DHC levels. Exogenous cholesterol supplementation directly addresses the primary biochemical deficiency. This specific regimen was evaluated in Phase 2 clinical trials led by Dr. Forbes D. Porter at the National Institutes of Health (NIH).

Brand names

OraPlus

Other names

OraPlus-Forbes Porter, M.D.-Smith-Lemli-Opitz syndromeSimvastatin Susp. + OraPlus (cholesterol)