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Article

Molecular Mechanisms and Therapeutics for SBMA/Kennedy's Disease

1 Oct 2019

Abstract excerpt

Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disease caused by a polyglutamine (polyQ) expansion in the androgen receptor (AR). Despite the fact that the monogenic cause of SBMA has been known for nearly 3 decades, there is no effective treatment for this disease, underscoring the complexity of the pathogenic mechanisms that lead to a loss of motor neurons and muscle in SBMA patients. In the...

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PMID: 31686397
DOI: 10.1007/s13311-019-00790-9

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