Creating an automated contemporaneous cohort in sickle cell anemia to predict survival after disease-modifying therapy

The Food and Drug Administration requires contemporaneous controls to compare clinical outcomes for participants receiving experimental gene therapy or gene editing clinical trials. However, developing a contemporaneous cohort of rare diseases requires multiple person-hours. In a single referral cen...

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Detalles Bibliográficos
Autores principales: Cronin, Robert M., Wuichet, Kristin, Ghafuri, Djamila L, Hodges, Brock, Chopra, Maya, He, Jing, Niu, Xinnan, Kassim, Adetola A., Wilkerson, Karina, Rodeghier, Mark, DeBaun, Michael R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2022
Materias:
Health Services and Outcomes
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393740/
https://www.ncbi.nlm.nih.gov/pubmed/36350716
http://dx.doi.org/10.1182/bloodadvances.2022008692

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10393740/
https://www.ncbi.nlm.nih.gov/pubmed/36350716
http://dx.doi.org/10.1182/bloodadvances.2022008692

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