Open-label trial of ranolazine for the treatment of paramyotonia congenita.

Autor: Lorusso S; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA., Kline D; Center for Biostatistics, Department of Biomedical Informatics, The Ohio State University, Columbus, Ohio, USA., Bartlett A; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA., Freimer M; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA., Agriesti J; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA., Hawash AA; Department of Neuroscience, Cell Biology and Physiology, Wright State University, Dayton, Ohio, USA., Rich MM; Department of Neuroscience, Cell Biology and Physiology, Wright State University, Dayton, Ohio, USA., Kissel JT; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA., David Arnold W; Division of Neuromuscular Disorders, Department of Neurology, The Ohio State University Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA.
Jazyk: angličtina
Zdroj: Muscle & nerve [Muscle Nerve] 2019 Feb; Vol. 59 (2), pp. 240-243. Date of Electronic Publication: 2018 Dec 21.
DOI: 10.1002/mus.26372
Abstrakt: Introduction: Paramyotonia congenita (PMC) is a nondystrophic myotonic disorder that is believed to be caused by a defect in Na v 1.4 sodium channel inactivation. Ranolazine, which acts by enhancing slow inactivation of sodium channels, has been proposed as a therapeutic option, but in vivo studies are lacking.
Methods: We conducted an open-label, single-center trial of ranolazine to evaluate efficacy and tolerability in patients with PMC. Subjective symptoms of stiffness, weakness, and pain as well as clinical and electrical myotonia were evaluated. Baseline measures were compared with those after 4 weeks of treatment with ranolazine.
Results: Ranolazine was tolerated well without any serious adverse events. Both subjective symptoms and clinical myotonia were significantly improved. Duration of myotonia was reduced according to electromyography, but this change was not statistically significant in all tested muscles.
Discussion: Our findings support the use of ranolazine as a treatment for myotonia in PMC and suggest that a randomized, placebo-controlled trial is warranted. Muscle Nerve 59:240-243, 2019.
(© 2018 Wiley Periodicals, Inc.)
Databáze: MEDLINE
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