Doctors inject gene therapy directly into Children's brains to fight rare fatal disease
NCT ID NCT01801709
Summary
This early-stage study tested a new gene therapy approach for children with a rare, severe brain disease called metachromatic leukodystrophy (MLD). Doctors injected a modified virus carrying a healthy gene directly into the brains of five young children (ages 6 months to 4 years) to see if it was safe and could slow the disease's progression. The main goal was to see if this one-time treatment could stop the neurological damage caused by MLD.
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Contacts and locations
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Locations
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Bicêtre Hospital - Paris Sud
Le Kremlin-Bicêtre, France
Conditions
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