Experimental gene therapy for rare neurodegenerative disease GAN shows promising results in NIH clinical trial.
An experimental gene therapy for giant axonal neuropathy (GAN), a rare, neurodegenerative disease without current treatment, showed promising results in a clinical trial led by the National Institutes of Health (NIH). The gene therapy, delivered directly into the spinal fluid using a modified virus, slowed motor function decline and regenerated sensory nerves in some patients. Further research will evaluate its effectiveness in younger children or earlier stages of the disease.
March 20, 2024
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