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

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


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.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Cardiovascular Agents / therapeutic use*
  • Electromyography
  • Female
  • Follow-Up Studies
  • Hand Strength
  • Humans
  • Male
  • Middle Aged
  • Myotonia Congenita / drug therapy*
  • Myotonia Congenita / physiopathology
  • Pilot Projects
  • Ranolazine / therapeutic use*
  • Self Report
  • Severity of Illness Index
  • Treatment Outcome
  • Young Adult


  • Cardiovascular Agents
  • Ranolazine