Lamotrigine, a drug commonly used to treat epilepsy and certain mood disorders, has been shown to be an excellent treatment option for a rare genetic neuromuscular disease known as non-dystrophic myotonia, in a recent trial.
Lamotrigine, a drug commonly used to treat epilepsy and certain mood disorders, has been shown to be an excellent treatment option for a rare genetic neuromuscular disease known as non-dystrophic myotonia, in a world-first trial led by UCL researchers.
Patients were randomly assigned to receive either mexiletine for eight weeks followed by lamotrigine for eight weeks, or the reverse order, with a seven-day break in between treatments. Neither the participants nor the researchers knew which treatment was being given at any time. In 2012, the same UCL team led a multi-centred international trial that repurposed mexiletine -- a sodium channel blocker -- and showed it was effective in treating non-dystrophic myotonia and improving quality of life.However, not all patients respond to the treatment and one third developed significant side-effects, the most common being reflux or gastrointestinal side effects. Additionally, mexiletine can't be prescribed during pregnancy, when myotonia often worsens.
This research will impact clinical practice globally as mexiletine is often not accessible in developing countries or is expensive in developed countries. The results demonstrate that lamotrigine is a comparable treatment and therefore provides an excellent treatment option for affected patients. Vinojini Vivekanandam, Iwona Skorupinska, Dipa L Jayaseelan, Emma Matthews, Richard J Barohn, Michael P McDermott, Michael G Hanna.Research has shown for the first time that the effects of Alagille syndrome, an incurable genetic disorder that affects the liver, could be reversed with a single drug. The study has the potential to ...
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