Scientists seek clues in rare genetic disorders to pave way for future treatments

NCT ID NCT05047354

Summary

This study aims to better understand Smith-Lemli-Opitz syndrome and related rare genetic disorders that affect how the body makes cholesterol. Researchers will observe up to 250 people with these conditions and their relatives over several years, collecting health information and samples. The goal is to gather detailed knowledge about how these diseases progress, which will help scientists design and test future treatments.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.