Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease

Interest in drug development for rare diseases has expanded dramatically since the Orphan Drug Act was passed in 1983, with 40% of new drug approvals in 2019 targeting orphan indications. However, limited quantitative understanding of natural history and disease progression hinders progress and incr...

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Detalles Bibliográficos
Autores principales: Mullin, Ariana P., Corey, Diane, Turner, Emily C., Liwski, Richard, Olson, Daniel, Burton, Jackson, Sivakumaran, Sudhir, Hudson, Lynn D., Romero, Klaus, Stephenson, Diane T., Larkindale, Jane
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7877853/
https://www.ncbi.nlm.nih.gov/pubmed/32702147
http://dx.doi.org/10.1111/cts.12845

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