Abstract
AbstractMexiletine is well-established sodium channel blocker that acts on cardiac myocytes and neurons. It has recently been repositioned as an orphan drug in the treatment of rare neuromuscular diseases in adults with nondystrophic myotonia. It has also long been used in some rare pediatric diseases in the areas of cardiopathy, epilepsy, neuromuscular diseases, and pain disorders. Here, we review the different uses of mexiletine reported in pediatrics, stating indications, efficacy, and tolerance. Special attention by health authorities to maintain access to mexiletine in rare pediatric diseases and further pediatric research in these rare syndromes are required.
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