Article
Minimum effective betamethasone dosage on the neurological phenotype in patients with ataxia-telangiectasia: a multicenter observer-blind study.
European journal of neurology - 1 Jun 2018
Cirillo E, Del Giudice E, Micheli R, Cappellari A M, Soresina A, Dellepiane R M, Pietrogrande M C, Dell'Era L, Specchia F, Pession A, Plebani A, Pignata C
Abstract excerpt
BACKGROUND AND PURPOSE: Ataxia-telangiectasia (A-T) is a rare neurodegenerative disease, due to A-T mutated (ATM) gene mutations, which typically presents with signs of progressive neurological dysfunction, cerebellar ataxia and uncoordinated movements. A-T severely affects patients' quality of life. Successful treatment options are still not available. The aim of this multicenter study, performed with a blind...
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