Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial.

Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schädelin S, Bieri O, Fischer D.
Trials. 2019. 20(1), 637.
Protocol of a multicenter, randomized, double-blind, placebo-controlled phase III trial study aiming to demonstrate safety and efficacy of tamoxifen over placebo in pediatric patients with DMD.

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Keywords: 6-min walk test; Duchenne muscular dystrophy; Motor function measure; Quantitative muscle MRI; Randomized placebo-controlled trial; Tamoxifen

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