Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH ...

A n investigational gene therapy, dubbed scAAV9/JeT-GAN, for a rare neurodegenerative disease, giant axonal neuropathy (GAN), that begins in early childhood was well tolerated and showed signs of ...