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Article

The ties that bind: functional clusters in limb-girdle muscular dystrophy

29 Jul 2020

Abstract excerpt

The limb-girdle muscular dystrophies (LGMDs) are a genetically pleiomorphic class of inherited muscle diseases that are known to share phenotypic features. Selected LGMD genetic subtypes have been studied extensively in affected humans and various animal models. In some cases, these investigations have led to human clinical trials of potential disease-modifying therapies, including gene replacement strategies for...

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PMID: 32727611
DOI: 10.1186/s13395-020-00240-7

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