3 resultados para Morgan, John Hunt, 1825-1864
em DI-fusion - The institutional repository of Université Libre de Bruxelles
Resumo:
Paris
Resumo:
info:eu-repo/semantics/published
Resumo:
Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We investigated the efficacy and safety of drisapersen, a 2'-O-methyl-phosphorothioate antisense oligonucleotide, given for 48 weeks.