Neurology 2021 01 12;96(2):e228-e240. Epub 2020 Oct 12.
From the Department of Neurology (C.H., E.L., S.R., K.E., W.M., J.H., J.D., N.D., C.Z., R.T., G.S., C.A.B., J.F.R., W.Z., C.T., M.P.M., R.M.), University of Rochester, MN; Virginia Commonwealth University (N.J.), Richmond; and Wake Forest Baptist Health (A.P.), Winston-Salem, NC.
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 performed a randomized, double-blind, placebo-controlled trial of mexiletine (150 mg 3 times daily) to evaluate its efficacy and safety in a homogenous cohort of adult ambulatory patients with DM1. The primary outcome was change in 6-minute walk distance at 6 months. Read More