Mexiletine for Treatment of Myotonia

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منابع مشابه

Mexiletine for treatment of myotonia: a trial triumph for rare disease networks.

PATIENTS WITH NONDYSTROPHIC MYOTONIAS TYPIcally have myotonia as an isolated symptom, without muscular wasting, although the patient’s myotonia may be associated with muscle weakness and fatigue or transient attacks of paralysis. Episodes of myotonia may be triggered by cold (paramyotonia congenita), potassium (potassium aggravated myotonia), or exercise (Thompson and Becker myotonia). Most cas...

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

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...

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Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach; study rationale and protocol

BACKGROUND To obtain evidence for the clinical and cost-effectiveness of treatments for patients with rare diseases is a challenge. Non-dystrophic myotonia (NDM) is a group of inherited, rare muscle diseases characterized by muscle stiffness. The reimbursement of mexiletine, the expert opinion drug for NDM, has been discontinued in some countries due to a lack of independent randomized controll...

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ژورنال

عنوان ژورنال: JAMA

سال: 2012

ISSN: 0098-7484

DOI: 10.1001/jama.2012.12906