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Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022

BACKGROUND: Compassionate use is a system that provides patients with expedited access to drugs which has not yet been approved, but currently in clinical trials. The investigational drugs have been authorized for compassionate use in cases involving patients suffered from life-threatening diseases...

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Detalles Bibliográficos
Autores principales: Wu, Jiayu, Yang, Yang, Yu, Jiaxin, Qiao, Luyao, Zuo, Wei, Zhang, Bo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10685565/
https://www.ncbi.nlm.nih.gov/pubmed/38017575
http://dx.doi.org/10.1186/s13023-023-02978-x