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Scientists investigate rare genetic disorder and cholesterol treatment

NCT ID NCT00001721

First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 27 times

Summary

This study looked at Smith-Lemli-Opitz syndrome (SLOS), a rare genetic disorder that affects how the body makes cholesterol and causes birth defects and intellectual disabilities. Researchers studied 130 patients and their mothers to better understand the disease, its complications, and whether adding cholesterol to the diet could help with growth and development. The goal was to gather knowledge, not to test a cure.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.