Inhibition of glutamate dehydrogenase as a new therapeutic approach in DMD

European and Chinese researchers have focused on the glutamate pathway as a possible therapeutic target in Duchenne muscular dystrophy (DMD):

• this molecule plays an important role in the presynaptic endings of neuromuscular junctions,
• pharmacological inhibition of the enzyme GLUD-1 (glutamate dehydrogenase 1) by compound R162 was analysed in mdx mice, a model of DMD,
• the compound R162 was shown to remodel neuromuscular junctions and improve muscle performance in mice.

This is the first time it has been studied in the mouse model of DMD, as a prelude to possible clinical trials.

 

Pharmacologic Inhibition of Glutamate Dehydrogenase 1 Improves Functional Recovery of Neuromuscular Junctions and Muscle Function in Duchenne Muscular Dystrophy. Pereira-Nunes A, Ammarah U, Shang M et al. Am J Pathol. 2025 Jun