Why the study?
Mexiletine has long been used in rare pediatric diseases across cardiology, neurology, and pain disorders, but a review of its reported pediatric uses, efficacy, and tolerance was needed.
This review highlights the diverse uses of mexiletine in rare pediatric conditions and advocates for continued access and research.
Mexiletine 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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Sarret et al. (2024) studied this question.
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