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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Bibliografiske detaljer
Udgivet i:Ann Neurol
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2016
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Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5064753/
https://ncbi.nlm.nih.gov/pubmed/26573217
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24555
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