ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder
加速计划:一项以患者为主导的自然史研究设计,旨在推动罕见病临床和治疗方面的发现
期刊:Cell Reports Medicine
影响因子:10.6
doi:10.1016/j.xcrm.2020.100158
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