Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle loss and premature death, most commonly from respiratory or cardiac failure. DMD is primarily caused by whole exon deletions, resulting in a shift of the dystrophin mRNA...

Descripción completa

Detalles Bibliográficos
Autores principales: Kinane, T. Bernard, Mayer, Oscar H., Duda, Petra W., Lowes, Linda P., Moody, Stephanie L., Mendell, Jerry R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2018
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5836407/
https://www.ncbi.nlm.nih.gov/pubmed/29278896
http://dx.doi.org/10.3233/JND-170272

Ejemplares similares

Ejemplares similares