Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial

Duchenne muscular dystrophy (DMD) is a progressive, lethal neuromuscular disorder caused by the absence of dystrophin protein due to mutations of the dystrophin gene. Drisapersen is a 2′-O-methyl-phosphorothioate oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA to restore the reading...

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Detalhes bibliográficos
Main Authors: Flanigan, Kevin M., Voit, Thomas, Rosales, Xiomara Q., Servais, Laurent, Kraus, John E., Wardell, Claire, Morgan, Allison, Dorricott, Susie, Nakielny, Joanna, Quarcoo, Naashika, Liefaard, Lia, Drury, Tom, Campion, Giles, Wright, Padraig
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4145871/
https://ncbi.nlm.nih.gov/pubmed/24321374
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2013.09.004
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