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Rare disease study aims to track MLIV's natural course

NCT ID NCT01067742

First seen Feb 01, 2026 · Last updated May 25, 2026 · Updated 19 times

Summary

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future clinical trials, not to test a treatment.

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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

  • Baylor Institute of Metabolic Disease

    Dallas, Texas, 75226, United States

Conditions

Explore the condition pages connected to this study.