Applicability of the International Cooperative Ataxia Rating Scale (ICARS) in younger patients and development of global rating instruments for patients with Ataxia Telangiectasia (AT)

Ataxia Telangiectasia (AT) is a severe neurologic disorder with progressive loss of walking capacity, ultimately leading to wheel chair. There is no curative treatment for AT, but some several attempts were made to use different drugs for improving neurologic symptoms. However, all attempts to check the benefit of any drug treatment  is hindered by the lack of  rating scale that measures the degree of neurologic disease in children with AT. The goal of our study is to validate the ICARS scale, in order to be further used in interventional studies with drugs treatments that might improve neurologic symptoms.