Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.
| Data(s) |
01/10/2014
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| 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. SCOPUS: ar.j info:eu-repo/semantics/published |
| Formato |
1 full-text file(s): application/pdf |
| Identificador |
uri/info:doi/10.1016/S1474-4422(14)70195-4 uri/info:pii/S1474-4422(14)70195-4 uri/info:pmid/25209738 https://dipot.ulb.ac.be/dspace/bitstream/2013/185496/1/Elsevier_169123.pdf http://hdl.handle.net/2013/ULB-DIPOT:oai:dipot.ulb.ac.be:2013/185496 |
| Idioma(s) |
en |
| Direitos |
1 full-text file(s): info:eu-repo/semantics/restrictedAccess |
| Fonte |
Lancet neurology, 13 (10 |
| Palavras-Chave | #Sciences bio-médicales et agricoles #Adrenal Cortex Hormones -- therapeutic use #Anti-Inflammatory Agents -- therapeutic use #Area Under Curve #Child #Child, Preschool #Double-Blind Method #Dystrophin -- genetics #Exons #Female #Gene Deletion #Humans #Male #Muscular Dystrophy, Duchenne -- drug therapy -- genetics #Oligonucleotides -- adverse effects -- therapeutic use #Real-Time Polymerase Chain Reaction #Treatment Outcome #Walking |
| Tipo |
info:eu-repo/semantics/article info:ulb-repo/semantics/articlePeerReview info:ulb-repo/semantics/openurl/article |
