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Expanding Approved Patient Populations for Rare Disease Treatment Using In Vitro Data
In vitro cell‐based data can be used to support the extension of pharmaceutical approval to patient subsets with unique genetic variants. A set of conditions should be satisfied to support the extension of approval. The disease mechanism should be well described, and the impact of variants on protei...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293129/ https://www.ncbi.nlm.nih.gov/pubmed/34496049 http://dx.doi.org/10.1002/cpt.2414 |