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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Podrobná bibliografie
Vydáno v:Medicine (Baltimore)
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: Wolters Kluwer Health 2019
Témata:
Research Article
On-line přístup: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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