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Article

Therapies for RYR1-Related Myopathies: Where We Stand and the Perspectives.

Current pharmaceutical design - 1 Jan 2022

Beaufils Mathilde, Travard Lauriane, Rendu John, Marty Isabelle

Abstract excerpt

RyR1-related myopathies are a family of genetic neuromuscular diseases due to mutations in the RYR1 gene. No treatment exists for any of these myopathies today, which could change in the coming years with the growing number of studies dedicated to the pre-clinical assessment of various approaches, from pharmacological to gene therapy strategies, using the numerous models developed up to now. In addition, the...

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Identifiers

PMID: 34514983
DOI: 10.2174/1389201022666210910102516

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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