Journal article
Improved muscle function in duchenne muscular dystrophy through l-arginine and metformin: An investigator-initiated, open-label, single-center, proof-of-concept-study
P Hafner, U Bonati, B Erne, M Schmid, D Rubino, U Pohlman, T Peters, E Rutz, S Frank, C Neuhaus, S Deuster, M Gloor, O Bieri, A Fischmann, M Sinnreich, N Gueven, D Fischer
Plos One | Published : 2016
Abstract
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 ..
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Awarded by Schweizerischer Nationalfonds zur Förderung der Wissenschaftlichen Forschung
Funding Acknowledgements
PH and UB were supported by grants of the University of Basel (https://medizin.unibas.ch/lehre/nachwuchsfoerderung.html) DF was supported by the Lorenzo-Piaggio Foundation, Switzerland; the Thomi-Hopf-Stiftung, Switzerland (www.thomi-hopf-stiftung.ch); the Neuromuscular Research Association Basel, Switzerland (http://www.nerab.org/); the University of Basel Children's Hospital, and the Department of Neurology, University Hospital Basel. The Department of Diagnostic and Interventional Radiology (AF) is supported by a grant from Bracco Suisse SA (http://imaging.bracco.com/ch-en/bracco-suisse-sa). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.