Improved mobility with metformin in patients with myotonic dystrophy type 1: a randomized controlled trial

Brain : a Journal of Neurology
Guillaume BassezMarc Peschanski

Abstract

Metformin, the well-known anti-diabetic drug, has been shown recently to improve the grip test performance of the DMSXL mouse model of myotonic dystrophy type 1. The drug may have positively affected muscle function via several molecular mechanisms, on RNA splicing, autophagia, insulin sensitivity or glycogen synthesis. Myotonic dystrophy remains essentially an unmet medical need. Since metformin benefits from a good toxicity profile, we investigated its potential for improving mobility in patients. Forty ambulatory adult patients were recruited consecutively at the neuromuscular reference centre of Henri-Mondor Hospital. Participants and investigators were all blinded to treatment until the end of the trial. Oral metformin or placebo was provided three times daily, with a dose-escalation period over 4 weeks up to 3 g/day, followed by 48 weeks at maximum dose. The primary outcome was the change in the distance walked during the 6-minute walk test, from baseline to the end of the study. Concomitant changes in muscle strength and effect on myotonia, gait variables, biological parameters and quality of life were explored. Patients randomized into two arms eventually revealed similar results in all physical measures and in the mean...Continue Reading

Citations

Jan 8, 2019·Brain : a Journal of Neurology·Vanessa Carvalho, João Martins
Jan 10, 2019·Médecine sciences : M/S·Cécile Martinat, Marc Peschanski
Jan 31, 2019·Journal of Cachexia, Sarcopenia and Muscle·John E Morley
Apr 4, 2019·Médecine sciences : M/S·Emmanuelle Campana-SalortGéraldine Honnet
Jun 22, 2019·Expert Review of Clinical Pharmacology·Federica RicciTiziana Mongini
Nov 22, 2019·International Journal of Cancer. Journal International Du Cancer·Rotana AlsaggafShahinaz M Gadalla
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Feb 26, 2019·Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology·Giuseppe VitaSonia Messina
Nov 23, 2019·Proceedings of the National Academy of Sciences of the United States of America·Ariadna BargielaRuben Artero
Dec 19, 2019·Frontiers in Neurology·Sylvia NieuwenhuisJeffrey C Glennon
Jun 5, 2019·Orphanet Journal of Rare Diseases·Marie De AntonioGuillaume Bassez
Nov 14, 2019·International Journal of Molecular Sciences·Arturo López CastelRubén Artero
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Dec 29, 2021·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·Sara BortolaniGiorgio Tasca

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