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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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