Rare disease study maps a path for future treatments
NCT ID NCT01067742
Summary
This study aimed to understand the natural progression of Mucolipidosis Type IV (MLIV), a rare genetic disorder that affects brain development and vision. It involved 7 participants with a confirmed diagnosis of MLIV. The goal was to gather detailed information about the disease to help design and measure the success of future potential treatments.
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Contacts and locations
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Locations
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Baylor Institute of Metabolic Disease
Dallas, Texas, 75226, United States
Conditions
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