Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study

Patricia Hafner, Ulrike Bonati, Beat Erne, Maurice Schmid, Daniela Rubino, Urs Pohlman, [...view 10 more...], Dirk Fischer


Altered neuronal nitric oxide synthase function in Duchenne muscular dystrophy leads to impaired mitochondrial function which is thought to be one cause of muscle damage in this disease. The study tested if increased intramuscular nitric oxide concentration can improve mitochondrial energy metabolism in Duchenne muscular dystrophy using a novel therapeutic approach through the combination of L-arginine with metformin. Five ambulatory, genetically confirmed Duchenne muscular dystrophy patients aged between 7–10 years were treated with L-arginine (3 x 2.5 g/d) and metformin (2 x 250 mg/d) for 16 weeks. Treatment effects were assessed using mitochondrial protein expression analysis in muscular biopsies, indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, and clinical scores of muscle performance. There were no serious side effects and no patient dropped out. Muscle biopsy results showed pre-treatment a significantly reduced mitochondrial protein expression and increased oxidative stress in Duchenne muscular dystrophy patients compared to controls. Post-treatment a significant elevation of proteins of the mitochondrial electron transport chain was observed as well as a reduction in oxidative stress. Treatment also decreased resting energy expenditure rates and energy substrate use shifted from carbohydrates to fatty acids. These changes were associated with improved clinical scores. In conclusion pharmacological stimulation of the nitric oxide pathway leads to improved mitochondria function and clinically a slowing of disease progression in Duchenne muscular dystrophy. This study shall lead to further development of this novel therapeutic approach into a real alternative for Duchenne muscular dystrophy patients

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My son have got GNC's  L-arginne 2 x 500mg per day for 2 years, I wish this method was helpful with his muscle.

Yanbin did you see any improvement in your son. He was given L-arginine only or metformin also, the above study I guess was with L-arginine and metformin both. Interested in know if I can start giving to my son.

Yes very interesting. Moein can you provide info on where this trial was performed? My son already takes Metformin, but only to counteract weight gain side effects of steroids.
David said:

Yes very interesting. Moein can you provide info on where this trial was performed? My son already takes Metformin, but only to counteract weight gain side effects of steroids.

I had given my son this combination before he was enrolled in Myostatin trial for Pfizer because it is not allowed in the trial

I think that there was an improvement and my son was having the courage to do tasks he was not doing it before,also he was less getting tired,more energy.

I am not sure if this was a placebo effect,but I think this combination has something to do

Moen how many days did you give this combination? Did you see any side effects. Did you give the dose as         L-arginine (3 x 2.5 g/d) and metformin (2 x 250 mg/d). Which brand of L-arginine you used? My son is 10 years old and not on any trial so far, hence I am very keen to start this.

For four months.I gave him just 1000 mg of L-Arginine with 250 mg Glucophage.
Brand Nowfood

Rahul, My son's doctor told me that prednisone is very important, it can extend child's ambulatory ability and prevents child's spine bend. other aux drugs just offer some auxliary effects,L-arginine too.But ,this kind of amino acid at least has no some side effcts. Has your son begun to take prednisone? his age is old enough. for his good living,maybe you sould consider my advise.

By the way, Some DMD cinical experiments show Inspra is effective to eliminate child's muscle oedema. My son took it  for 25mg per day, He's more than 5 years old and 18kg weight now.

Yanbin, Thanks for your suggestion, I am giving my son Deflazacort for the last 6 months. Was considering giving L-Arginine and Metformin in addition as the results look promising.

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