Experimental gene therapy offers hope for rare nerve disease
NCT ID NCT02362438
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 25 times
Summary
This study tested a gene transfer treatment for giant axonal neuropathy (GAN), a rare genetic disease that damages nerves and affects movement, breathing, and other functions. Fourteen people aged 3 and older with GAN received the treatment through a spinal tap. The main goal was to check safety, with researchers also looking for signs of improvement in nerve function. While not a cure, this approach aims to slow or control the disease.
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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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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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