Drug intelligence / Profile preview

cholesterol emulsion

Development stage
Phase 2
Lead developer
Universitätsklinikum Münster
Modality
Small Molecules
Administration
Oral
01

Overview

Cholesterol emulsion is a nutritional therapeutic formulation developed to treat Smith-Lemli-Opitz syndrome (SLOS), a rare autosomal recessive disorder characterized by impaired cholesterol biosynthesis. The formulation delivers cholesterol in an emulsion form to improve solubility and prevent the clumping and sedimentation typically associated with oil-based or aqueous cholesterol suspensions. By providing exogenous cholesterol, the therapy aims to increase total cholesterol levels and suppress the accumulation of the toxic precursor 7-dehydrocholesterol (7-DHC) through feedback inhibition of the cholesterol biosynthetic pathway. It has been evaluated in clinical studies led by researchers at the Universitätsklinikum Münster.

Other names
cholesterol
02

Targets

HMGCR (3-hydroxy-3-methylglutaryl-coenzyme A reductase)LDLR (Low-density lipoprotein receptor)

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