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.
Articles in scientific journals
Ropars J, Gravot F, Ben Salem D, Rousseau F, Brochard S, Pons C. Neurology. 2020. 94(3):117-133. Review to assess the evidence of a relationship between muscle MRI and disease severity in Duchenne muscular dystrophy (DMD). Correlations between motor function tests,...read more
Evaluation of Speed-Accuracy Trade-Off in a Computer Task to Identify Motor Difficulties in Individuals With Duchenne Muscular Dystrophy – A Cross-Sectional Study
da Silva TD, Ribeiro-Papa DC, Coe S, Malheiros SRP, Massetti T, Meira Junior CM, Nicolai Ré AH, Collett J, Monteiro CBM, Dawes H. Res Dev Disabil. 2020. 96:103541. The aim of the study was to evaluate movement time during a task at various levels of difficulty and to...read more
Voos MC, Goya PSA, de Freitas BL, Pires AMT, Favero FM, Caromano FA. Arch Physiother. 2020 18;10:4. doi: 10.1186/s40945-020-0074-3. eCollection 2020. The aim of the study was to describe the evolution of timed immersion expiration in 57 patients with muscular...read more
The Use of the Gait Profile Score and Gait Variable Score in Individuals With Duchenne Muscular Dystrophy
de Souza MA, Cezarani A, Lizzi EAD, Davoli GBQ, Mattiello SM, Jones R, Mattiello-Sverzut AC J Biomech. 2020. 98, 109485. Study of the applicability of the Gait Deviation Index (GDI) and the Gait Profile Score (GPS) in patients with duchenne Muscular Dystrophy, using...read more
Analysis of Different Device Interactions in a Virtual Reality Task in Individuals With Duchenne Muscular Dystrophy-A Randomized Controlled Trial
de Freitas BL, da Silva TD, Crocetta TB, Massetti T, de Araújo LV, Coe S, Dawes H, Caromano FA, Monteiro CBM. Front Neurol. 2019 Jan 29;10:24. The aim of the study was to compare performance of patients with Duchenne Muscular Dystrophy to perform a virtual task using...read more
Nagy S, Schädelin S, Hafner P, Bonati U, Scherrer D, Ebi S, Schmidt S, Orsini AL, Bieri O, Fischer D. Muscle Nerve. 2020. 61(1), 63-68. In this retrospective study, the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD...read more