Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D.
JAMA Netw Open. 2019. 2;2(10):e1914171.
Results of a single-center randomized clinical trial aiming to evaluate the benefits and harms of a combination of l-citrulline and metformin treatment among patients with DMD.
The primary end point was the change in MFM-D1 score from baseline to week 26.
Treatment with combination therapy was not associated with an overall reduction in motor function decline among ambulant patients with DMD; however, a reduction in motor function decline was observed among the stable subgroup of patients treated with combination therapy.

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