metformin + L-arginine

This combination therapy, consisting of the biguanide antidiabetic drug metformin and the amino acid L-arginine (or its precursor L-citrulline), is being investigated by the University Hospital Basel for the treatment of Duchenne muscular dystrophy (DMD). The therapeutic rationale addresses the displacement of neuronal nitric oxide synthase (nNOS) and subsequent mitochondrial dysfunction characteristic of DMD. Metformin acts as an AMP-activated protein kinase (AMPK) activator to enhance mitochondrial biogenesis and energy metabolism, while L-arginine and L-citrulline serve as substrates for nitric oxide (NO) production. By increasing intramuscular NO concentrations, the combination aims to improve muscle function, reduce oxidative stress, and delay disease progression. Clinical evaluation has progressed from a proof-of-concept study in ambulatory boys to randomized, placebo-controlled trials.

Other names

metformin + L-citrulline