Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: a randomized controlled trial.

ABSTRACT:

Context

Nondystrophic myotonias (NDMs) are rare diseases caused by mutations in skeletal muscle ion channels. Patients experience delayed muscle relaxation causing functionally limiting stiffness and pain. Mexiletine-induced sodium channel blockade reduced myotonia in small studies; however, as is common in rare diseases, larger studies of safety and efficacy have not previously been considered feasible.

Objective

To determine the effects of mexiletine for symptoms and signs of myotonia in patients with NDMs.

Design, setting, and participants

A randomized, double-blind, placebo-controlled 2-period crossover study at 7 neuromuscular referral centers in 4 countries of 59 patients with NDMs conducted between December 23, 2008, and March 30, 2011, as part of the National

SUBMITTER: Statland JM 

PROVIDER: S-EPMC3564227 | biostudies-literature | 2012 Oct

REPOSITORIES: biostudies-literature