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Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy

2025-01-27

Abstract excerpt

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease. There are currently few effective therapies to treat the disease, although many approaches are being pursued. Certain histone deacetylase inhibitors (HDACi) have been shown to ameliorate DMD phenotypes in mouse and zebrafish animal models, and the HDACi givinostat has recently gained FDA approval for DMD. Our goal was to identify additi...

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Identifiers and source

Literature Corpus work
f117eb54-8791-56c1-89e5-5d054c4c1bb5
DOI
10.1101/2025.01.24.634796
Open publication

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Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophyDOI 10.1101/2025.01.24.634796
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