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Rare disease study pulled before starting: scientists sought clues in cell stress

NCT ID NCT05514470

First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 39 times

Summary

This study was planned to learn how certain rare genetic mutations disrupt protein production and cause severe diseases affecting the lungs, liver, brain, and digestion. Researchers would have used cells from patients to measure protein levels and stress responses. However, the study was withdrawn before enrolling any participants, so no results are available.

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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

Locations

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

Conditions

The condition(s) this trial relates to.

liver disorder Neurologic Manifestations pancreas disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.