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Article

Longitudinal changes of outcome measures in spinal and bulbar muscular atrophy

6 Jul 2012

Abstract excerpt

Spinal and bulbar muscular atrophy is an adult-onset, hereditary motor neuron disease caused by the expansion of a trinucleotide CAG repeat within the gene encoding the androgen receptor. To date, several agents have been shown to prevent or slow disease progression in animal models of this disease. For the translational research of these agents, it is necessary to perform the detailed analysis of natural history...

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PMID: 22773541
DOI: 10.1093/brain/aws170

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