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NICE guidance - mexiletine for treating the symptoms of myotonia in non-dystrophic myotonic disorders

Authoring team

Mexiletine (Namuscla) is recommended, within its marketing authorisation, as an option for treating the symptoms of myotonia in adults with non-dystrophic myotonic disorders (1).

Mexiletine is a class 1b antiarrhythmic medication with a high affinity for muscle sodium channels (2)

In a series of N-of-1 trials of mexiletine vs placebo in patients with nondystrophic myotonia, there was a reduction in mean daily-reported muscle stiffness that was consistent with the treatment effect in a previous randomized clinical trial (3)

  • findings support the efficacy of mexiletine for treatment of nondystrophic myotonia as well as the feasibility of N-of-1 trials for assessing interventions in some chronic rare diseases

Reference:

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