Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

OBJECTIVE: To continue evaluation of the long‐term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three‐year progression of eteplirsen‐treated patients was compared to matched historical con...

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Detalles Bibliográficos
Autores principales: Mendell, Jerry R., Goemans, Nathalie, Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Shao, James, Kaye, Edward M., Mercuri, Eugenio, Hamid, Hoda Abdel, Byrne, Barry J., Connolly, Anne M., Dracker, Robert A., Matthew Frank, L., Heydemann, Peter T., O'Brien, Kevin C., Sparks, Susan E., Specht, Linda A., Rodino‐Klapac, Louise, Sahenk, Zarife, Al‐Zaidy, Samiah, Cripe, Linda H., Lewis, Sarah, M, Pane, E, Mazzone, S, Messina, GL, Vita, Bertini, D Amico A, Casimiro, Berardinelli A, Y, Torrente, F, Magri, GP, Comi, G, Baranello, T, Mongini, A, Pini, R, Battini, E, Pegoraro, C, Bruno, L, Politano, S, Previtali
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5064753/
https://www.ncbi.nlm.nih.gov/pubmed/26573217
http://dx.doi.org/10.1002/ana.24555

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5064753/
https://www.ncbi.nlm.nih.gov/pubmed/26573217
http://dx.doi.org/10.1002/ana.24555

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