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Rare disease SPLIS under the microscope: new registry launches

NCT ID NCT04885179

First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 27 times

Summary

This study aims to learn more about a rare condition called SPLIS, caused by a genetic enzyme deficiency. Researchers will collect medical records, genetic information, and samples like blood and urine from up to 120 participants of all ages. No treatments are given; the goal is to track survival and disease progression to better understand SPLIS.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • University of California San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

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

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

Conditions

Explore the condition pages connected to this study.