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Neurology. 2017 Aug 15;89(7):710-713. doi: 10.1212/WNL.0000000000004229. Epub 2017 Jul 14.

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

Author information

1
From the Department of Neurology (W.D.A., A.S., A.B., J.T.K.), The Ohio State University Wexner Medical Center; Center for Biostatistics (D.K.), Department of Biomedical Informatics, The Ohio State University, Columbus; and Neuroscience, Cell Biology, and Physiology and Neurology (A.A.H., K.R.N., M.M.R.), Wright State University, Dayton, OH. william.arnold@osumc.edu.
2
From the Department of Neurology (W.D.A., A.S., A.B., J.T.K.), The Ohio State University Wexner Medical Center; Center for Biostatistics (D.K.), Department of Biomedical Informatics, The Ohio State University, Columbus; and Neuroscience, Cell Biology, and Physiology and Neurology (A.A.H., K.R.N., M.M.R.), Wright State University, Dayton, OH.

Abstract

OBJECTIVE:

To determine open-label, pilot study whether ranolazine could improve signs and symptoms of myotonia and muscle stiffness in patients with myotonia congenita (MC).

METHODS:

Thirteen participants were assessed at baseline and 2, 4, and 5 weeks. Ranolazine was started after baseline assessment (500 mg twice daily), increased as tolerated after week 2 (1,000 mg twice daily), and maintained until week 4. Outcomes included change from baseline to week 4 in self-reported severity of symptoms (stiffness, weakness, and pain), Timed Up and Go (TUG), hand grip and eyelid myotonia, and myotonia on EMG.

RESULTS:

Self-reported severity of stiffness (p < 0.0001) and weakness (p < 0.01) was significantly improved compared with baseline. TUG and grip myotonia times were reduced (p = 0.03, p = 0.01). EMG of the abductor digiti minimi and tibialis anterior showed significantly reduced myotonia duration (p < 0.001, p < 0.01) at week 4. No participant discontinued ranolazine because of side effects.

CONCLUSIONS:

Ranolazine appeared to be well tolerated over a period of 4 weeks in individuals with MC, and ranolazine resulted in improvement of signs and symptoms of muscle stiffness. The findings of this study suggest that ranolazine should be investigated in a larger controlled study.

CLASSIFICATION OF EVIDENCE:

This study provides Class IV evidence that ranolazine improves myotonia in myotonia congenita.

PMID:
28710329
PMCID:
PMC5562961
[Available on 2018-08-15]
DOI:
10.1212/WNL.0000000000004229
[Indexed for MEDLINE]

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