Importance In rare diseases it is difficult to achieve high-quality evidence of treatment
efficacy because of small cohorts and clinical heterogeneity. With emerging treatments for …

Context Nondystrophic myotonias (NDMs) are rare diseases caused by mutations in skeletal
muscle ion channels. Patients experience delayed muscle relaxation causing functionally …

Objective To assess mexiletine's long-term safety and effect on 6-minute walk distance in a
well-defined cohort of patients with myotonic dystrophy type 1 (DM1). Methods We …

Objective: To determine if mexiletine is safe and effective in reducing myotonia in myotonic
dystrophy type 1 (DM1). Background: Myotonia is an early, prominent symptom in DM1 and …

Mexiletine is the only drug with proven effect for treatment of non-dystrophic myotonia, but
mexiletine is expensive, has limited availability and several side effects. There is therefore a …

Introduction: More than 90% of amyotrophic lateral sclerosis (ALS) patients have muscle
cramps, but evidence‐based treatments have not been available. Methods: A multicenter …

Objective: To determine the safety and tolerability of mexiletine in a phase II double-blind
randomized controlled trial of sporadic amyotrophic lateral sclerosis (SALS). Methods: Sixty …

Background Abnormal delayed relaxation of skeletal muscles, known as myotonia, can
cause disability in myotonic disorders. Sodium channel blockers, tricyclic antidepressive …

Methods| All patients with genetically confirmed nondystrophic myotonia or hyperkalemic
periodic paralysis prescribed mexiletine with a minimum of 6 months follow-up in our clinic …

Health Sciences, Centre de recherche Charles-Le-Moyne, Université de Sherbrooke, Centre
intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean, 2230 de …