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Dose-finding studies in drug development for rare genetic diseases

BACKGROUND: The small patient populations inherent to rare genetic diseases present many challenges to the traditional drug development paradigm. One major challenge is generating sufficient data in early phase studies to inform dose selection for later phase studies and dose optimization for clinic...

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Detalles Bibliográficos
Autores principales:	Wang, Lingshan, Wang, Jie, Feng, Ji, Doi, Mary, Pepe, Salvatore, Pacanowski, Michael, Schuck, Robert N.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8985255/ https://www.ncbi.nlm.nih.gov/pubmed/35382851 http://dx.doi.org/10.1186/s13023-022-02298-6

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