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Article

Natural history of LGMD 2A for delineating outcome measures in clinical trials

4 Mar 2016

Abstract excerpt

OBJECTIVE: Limb-girdle muscular dystophy 2A (LGMD2A, OMIM) is a slowly progressive myopathy caused by the deficiency in calpain 3, a calcium-dependent cysteine protease of the skeletal muscle. METHODS: In this study, we carried out an observational study of clinical manifestations and disease progression in genetically confirmed LGMD2A patients for up to 4 years. A total of 85 patients, aged 14-65 years, were...

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Share to Calpain Protease Function and Regulation
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PMID: 27081656
DOI: 10.1002/acn3.287

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