Article
Nonsense readthrough by ataluren prevents physical functional decline in aged dysferlinopathy mouse model
2026-06-03
Abstract excerpt
<title>Abstract</title> <p> Background Dysferlinopathy is a progressive muscular dystrophy frequently caused by nonsense variants in the dysferlin gene. Our previous study demonstrated that the nonsense readthrough agent ataluren improves physical function in an early-stage dysferlinopathy model of 9 weeks old. Its therapeutic impact on more advanced stages remains unanswered. This study evaluates the efficacy...
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Identifiers and source
- Literature Corpus work
- 88c4e551-befb-5614-93e4-b830f9966010
- DOI
- 10.21203/rs.3.rs-9819173/v1