Vitaminlike compound eases rare disorder
By Nathan Seppa
Researcher Salvatore DiMauro had never seen anything like it. An 11-year-old boy who had been wheelchair-bound for years was up and walking, thanks to some pills you can buy over the counter.
The patient had a hereditary syndrome called familial cerebellar ataxia. Like other ataxias, it arises from problems with the spinal cord and cerebellum, the part of the brain responsible for orchestrating nerve signals that coordinate muscles. This form of ataxia leaves patients with muscle damage, as well. They slowly lose the ability to walk, perform simple movements, and even talk.