Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model

BACKGROUND: Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory individuals aged two years and older. This study explo...

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Detalles Bibliográficos
Autores principales: Williams, Kate, Davidson, Ian, Rance, Mark, Boehnke, Axel, Buesch, Katharina, Acaster, Sarah
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8380095/
https://www.ncbi.nlm.nih.gov/pubmed/34420127
http://dx.doi.org/10.1186/s41687-021-00341-x

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