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GLUD1 Inhibitor R162 Restores Strength and Coordination in DMD Mouse Models

Systemic R162 treatment produced striking improvements in muscle performance, neuromuscular junction structure, with no detectable adverse effects.

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Overview

  • Researchers identified glutamate dehydrogenase 1 as a novel non-steroidal therapeutic target for Duchenne muscular dystrophy
  • In mdx mice, systemic R162 dosing led to significant gains in grip strength and motor coordination
  • R162 reprogrammed glutamate metabolism in dystrophic muscle, boosting satellite cell activity and restoring acetylcholine levels at neuromuscular junctions
  • Macrophage-mediated regeneration proved essential for R162’s full therapeutic effect on muscle function
  • Chronic R162 treatment was well tolerated with no impacts on body weight, food intake or behavior