Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
Status:
Completed
Trial end date:
2011-07-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to determine whether supplementation with an oil-based
cholesterol suspension will correct the biochemical abnormalities in cholesterol and its
precursors in individuals with the Smith-Lemli-Opitz syndrome.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital