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Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study

BACKGROUND: Expanded access programs (EAPs) allow patients with serious, life-threatening conditions access to drugs prior to their formal approval. Despite the possible benefits for patients, EAPs present several challenges, including uncertainty regarding a drug’s efficacy and safety as well as in...

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Detalles Bibliográficos
Autores principales:	Kiefer, Petra, Kirschner, Janbernd, Pechmann, Astrid, Langer, Thorsten
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2020
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7391692/ https://www.ncbi.nlm.nih.gov/pubmed/32727502 http://dx.doi.org/10.1186/s13023-020-01477-7

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