A new hope for patients with non-dystrophic myotonia

Lamotrigine as a viable treatment for rare muscle disorder.

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Lamotrigine, a drug used for epilepsy and mood disorders, was found to be a good treatment for a rare muscle disease called non-dystrophic myotonia.

In a first trial, UCL researchers compared lamotrigine with another drug, Mexiletine, to test their effectiveness in treating the condition. The study was published in Lancet Neurology.

In a trial at UCL, 60 adults with non-dystrophic myotonia were treated with two drugs, Mexiletine and lamotrigine, to see which worked better.

Patients were randomly given one drug for eight weeks and then switched to the other after a short break. Neither patients nor doctors knew which drug was used. Both drugs reduced muscle stiffness, the main symptom, by a similar amount.

Dr. Vino Vivekanandam, a neurologist at UCL, highlighted that many rare diseases, including neurological ones, lack treatments due to their rarity, making trials difficult.

Comparing drugs in trials helps find the best treatment. The recent trial results are essential for patients with non-dystrophic myotonias. This muscle disorder causes stiffness, pain, and fatigue, leading to disability. There’s no cure yet.

In 2012, a UCL-led trial showed that Mexiletine, a sodium channel blocker, was effective in treating non-dystrophic myotonia, making it the standard treatment. However, some patients don’t respond well or experience side effects, and Mexiletine can’t be used during pregnancy.

The new trial found that lamotrigine is a good alternative. It has fewer side effects, can be used in pregnancy, and is cheaper.

This research will have a global impact because Mexiletine is expensive or complicated to access in many countries. The study shows that lamotrigine is just as effective, offering a good alternative for patients.

Dr. Vivekanandam said, “They’ve already created a personalized treatment plan based on the trial, considering how drugs work and economic factors. “

Journal reference :

  1. Vinojini Vivekananda, Iwona Skorupinska, et al., Mexiletine versus lamotrigine in non-dystrophic myotonias: a randomized, double-blind, head-to-head, crossover, non-inferiority, phase 3 trial. Lancet Neurology. DOI: 10.1016/S1474-4422(24)00320-X.
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