New study tracks rare disease MLIV to unlock its secrets

NCT ID NCT05782387

First seen May 01, 2026 · Last updated May 11, 2026 · Updated 2 times

Summary

This study looks back at medical records of 50 people with mucolipidosis type IV (MLIV) to describe how the disease progresses. Researchers want to know when children reach or lose developmental milestones, how their movement and vision change, and what brain scans show. The goal is to better understand MLIV, which can help design future treatments and improve care.

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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

  • Massachusetts General Hospital

    Boston, Massachusetts, 02144, United States

Conditions

Explore the condition pages connected to this study.