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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The American Society of Hematology
2022
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Materias: | |
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 |
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