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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: 2013
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4145871/
https://www.ncbi.nlm.nih.gov/pubmed/24321374
http://dx.doi.org/10.1016/j.nmd.2013.09.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4145871/
https://www.ncbi.nlm.nih.gov/pubmed/24321374
http://dx.doi.org/10.1016/j.nmd.2013.09.004

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