A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment

BACKGROUND: Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in patients with Duchenne muscular dystrophy and confirmed exon-51 amenable genetic mutations. Chart review Study 4658-405 (405) further followed these patients while receiving eteplirsen during usual c...

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Detalles Bibliográficos
Autores principales: Mitelman, Olga, Abdel-Hamid, Hoda Z., Byrne, Barry J., Connolly, Anne M., Heydemann, Peter, Proud, Crystal, Shieh, Perry B., Wagner, Kathryn R., Dugar, Ashish, Santra, Sourav, Signorovitch, James, Goemans, Nathalie, McDonald, Craig M., Mercuri, Eugenio, Mendell, Jerry R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2022
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8842766/
https://www.ncbi.nlm.nih.gov/pubmed/34420980
http://dx.doi.org/10.3233/JND-210665

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8842766/
https://www.ncbi.nlm.nih.gov/pubmed/34420980
http://dx.doi.org/10.3233/JND-210665

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