First human trial offers hope for rare, fatal nerve disease
NCT ID NCT02362438
Summary
This is a first-in-human safety study testing a new gene therapy for Giant Axonal Neuropathy (GAN), a rare and fatal genetic disorder that damages nerves. Researchers are giving a single spinal injection that delivers a working copy of the faulty gene to the brain and spinal cord. The study aims to see if this treatment is safe and if it can slow or stop the progression of the disease in children and adults.
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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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