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Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives

Rare diseases represent a highly heterogeneous group of disorders with high phenotypic and genotypic diversity within individual conditions. Due to the small numbers of people affected, there are unique challenges in understanding rare diseases and drug development for these conditions, including pa...

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Detalles Bibliográficos
Autores principales:	Liu, Jing, Barrett, Jeffrey S., Leonardi, Efthimia T., Lee, Lucy, Roychoudhury, Satrajit, Chen, Yong, Trifillis, Panayiota
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Supplement Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10107901/ https://www.ncbi.nlm.nih.gov/pubmed/36461748 http://dx.doi.org/10.1002/jcph.2134

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