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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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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 |