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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Bibliografische gegevens
Gepubliceerd in:Medicine (Baltimore)
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Wolters Kluwer Health 2019
Onderwerpen:
Research Article
Online toegang: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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