This rare genetic disorder may affect fewer than 100 people worldwide. You may experience difficulty walking or numbness in your arms and legs at first, but it can develop into more serious symptoms.

Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of symptoms and a challenging prognosis. Now, a study published recently in the New ...

Gene therapy delivered for the first time via lumbar puncture showed promise of benefit in slowing progression of giant axonal neuropathy (GAN) in a phase I clinical trial. The slope of change in ...

Research group from the University of Helsinki, Finland, has identified a new disease gene for early-onset axonal neuropathy and mild intellectual disability through an international research network, ...

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 ...

A new Guillain-Barré syndrome mimic that appeared to be triggered by mild infection emerged in children with biallelic RCC1 variants, a case series showed. One of the first cases was seen in a ...