Experimental enzyme treatment tested on single child with devastating brain disease
NCT ID NCT01303146
Summary
This study tested an experimental enzyme replacement therapy for a child with late infantile MLD, a rare and severe brain disease. The child had previously received a stem cell transplant but still needed additional treatment. Researchers gave intravenous enzyme infusions every other week for 18 months to see if it could help preserve nerve function and slow disease progression.
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Contacts and locations
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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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