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Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9-month follow-up in a case report

D’Ambrosio P, Orsini C, Nigro V, Politano L. Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9-month follow-up in a case report. Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology 2018;37:272-274   Abstract Duchenne muscular Dystrophy (DMD) is a […]