An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH).
Experimental gene therapy for giant axonal neuropathy shows promise in clinical trial retrieved 21 March 2024 from https://medicalxpress.com/news/2024-03-experimental-gene-therapy-giant-axonal.html
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