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National Institute of Health (NIH)

Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial

Treatment for rare childhood disease was well tolerated and slowed loss of motor function 20 March 2024 -- Maryland, US -- 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...