ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder

Geographically dispersed patients, inconsistent treatment tracking, and limited infrastructure slow research for many orphan diseases. We assess the feasibility of a patient-powered study design to overcome these challenges for Castleman disease, a rare hematologic disorder. Here, we report initial...

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Detalles Bibliográficos
Autores principales: Pierson, Sheila K., Khor, Johnson S., Ziglar, Jasira, Liu, Amy, Floess, Katherine, NaPier, Erin, Gorzewski, Alexander M., Tamakloe, Mark-Avery, Powers, Victoria, Akhter, Faizaan, Haljasmaa, Eric, Jayanthan, Raj, Rubenstein, Arthur, Repasky, Mileva, Elenitoba-Johnson, Kojo, Ruth, Jason, Jacobs, Bette, Streetly, Matthew, Angenendt, Linus, Patier, Jose Luis, Ferrero, Simone, Zinzani, Pier Luigi, Terriou, Louis, Casper, Corey, Jaffe, Elaine, Hoffmann, Christian, Oksenhendler, Eric, Fosså, Alexander, Srkalovic, Gordan, Chadburn, Amy, Uldrick, Thomas S., Lim, Megan, van Rhee, Frits, Fajgenbaum, David C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7762771/
https://www.ncbi.nlm.nih.gov/pubmed/33377129
http://dx.doi.org/10.1016/j.xcrm.2020.100158

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