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Article

Unmet Needs and Evolving Treatment for Limb Girdle Muscular Dystrophies

2 Sept 2021

Abstract excerpt

Limb-girdle muscular dystrophies (LGMDs) represent a major group of muscle disorders. Treatment is sorely needed and currently expanding based on safety and efficacy adopting principles of single-dosing gene therapy for monogenic autosomal recessive disorders. Gene therapy has made in-roads for LGMD and this review describes progress that has been achieved for these conditions. This review first provides a...

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PMID: 34472379
DOI: 10.2217/nmt-2020-0066

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