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

ABSTRACT:

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 ameliorate dystrophic pathology, but prior strategies have been hindered by inadequate efficacy and off-target effects.

Methods

Comparative transcriptomic analyses were conducted to assess concordance between gene expression profiles induced by direct AMPK activation and those observed in DMD patient muscle. To evaluate the therapeutic potential of sustained AMPK activ

SUBMITTER: Ng SY 

PROVIDER: S-EPMC12872333 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature