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Article

Direct AMPK Activation Confers Mutation-Independent Therapeutic Benefit in Duchenne Muscular Dystrophy.

Journal of cachexia, sarcopenia and muscle - 1 Feb 2026

Ng Sean Y, Mikhail Andrew I, Mattina Stephanie R, Lesinski Magda A, Rebalka Irena A, Hamstra Sophie I, Xhuti Donald, Fajardo Val A, Tarnopolsky Mark A, Nederveen Joshua P, Steinberg Gregory R, Hawke Thomas J, Ljubicic Vladimir

Abstract excerpt

BACKGROUND: Duchenne muscular dystrophy (DMD) is a severe, life-limiting neuromuscular disorder (NMD) characterized by progressive muscle wasting and mitochondrial dysfunction. Although gene therapies offer promise, even those already approved by regulatory agencies, their use remains constrained by mutation specificity, delivery challenges and durability. Pharmacologically targeting AMPK has shown potential to...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 41638771
DOI: 10.1002/jcsm.70200
PMCID: PMC12872333

Genes

DMD

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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