Preserving the ability to swallow reduces the need for invasive interventions and lowers the risk of life-threatening ...

An 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 ...

Learn more about assistive devices for children with SMA, find out who will be part of your care team, and test your knowledge about the condition.

Gene activity in SMA may be dysregulated, but the way the disease affects genes seems to vary across types of tissues, per a ...

The team at King's College London used gene therapy to repair damage in the spinal cord of rats. The animals could then pick up and eat sugar cubes with their front paws. It is early stage ...