Experimental enzyme therapy tested in rare child brain disease after transplant
NCT ID NCT01303146
First seen Apr 01, 2026 · Last updated May 14, 2026 · Updated 5 times
Summary
This study tested an experimental enzyme therapy called METAZYM in one child with late infantile metachromatic leukodystrophy (MLD) who had already received a stem cell transplant. The goal was to see if the enzyme could slow nerve damage and improve function. The child received regular infusions of the drug, and researchers monitored nerve health, motor skills, and safety over time.
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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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Locations
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Department of Pediatric Endocrinology and Neurology, Saint Vincent de Paul Hospital
Paris, 75014, France
Conditions
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