Abstract
Introduction: DBMD is an X-linked disorder causing severe, progressive muscle loss in children. Patients (∼13%) have DBMD due to a nonsense (premature stop codon) mutation in the gene for dystrophin, a protein needed for muscle stability. Ataluren is an investigational drug designed to promote ribosomal readthrough of premature stop codons in mRNA, leading to production of full-length, functional protein. Methods: This Phase 2b, randomized, double-blind, placebo-controlled, dose-ranging study assessed the efficacy and safety of ataluren in males ⩾5year with nmDBMD documented by dystrophin gene sequencing.
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