Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutations amena...

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Udgivet i:Medicine (Baltimore)
Main Authors: Alfano, Lindsay N., Charleston, Jay S., Connolly, Anne M., Cripe, Linda, Donoghue, Cas, Dracker, Robert, Dworzak, Johannes, Eliopoulos, Helen, Frank, Diane E., Lewis, Sarah, Lucas, Karin, Lynch, Jessie, Milici, A. J., Flynt, Amy, Naughton, Emily, Rodino-Klapac, Louise R., Sahenk, Zarife, Schnell, Frederick J., Young, G. David, Mendell, Jerry R., Lowes, Linda P.
Format: Artigo
Sprog:Inglês
Udgivet: Wolters Kluwer Health 2019
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6617421/
https://ncbi.nlm.nih.gov/pubmed/31261494
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1097/MD.0000000000015858
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