This case study, published in Acta Myologica, details an ambulant 2-year-old patient with nonsense mutation Duchenne muscular dystrophy (nmDMD), who received early treatment with a mutation-specific therapy in an expanded-access program
Publication: Early treatment with ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy
MED-ALL-CORP-2200029 | September 2023
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