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A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial

Creator: Crow, Rebecca A, Hart, Kimberly A, McDermott, Michael P, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michele, Brown, Mary W, Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry B, Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan Y, Morandi, Lucia, Butterfield, Russell J, Horrocks, Iain, Roper, Helen, Flanigan, Kevin M, Kuntz, Nancy L, Mah, Jean K, Morrison, Leslie, Darras, Basil T, von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, McDonald, Craig M, Vita, Giuseppe, Barohn, Richard J, Finkel, Richard S, Wicklund, Matthew, McMillan, Hugh J, Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W., Howard, James F, Thangarajh, Mathula, Campbell, Craig, Griggs, Robert C, Bushby, Kate, Guglieri, Michela

Abstract Background Trials in rare diseases have many challenges, among which are the need to set up multiple sites in different countries to achieve recruitment targets and the divergent landscape of (more)

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13063_2018_article_2645.pdf

Collection: BioMed Central

Date Deposited: 2018-05-25

Date Created: 2018-05-25

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Additional file 1: Checklist supplementary information

Collection: BioMed Central

Date Deposited: 2018-05-25

Date Created: 2018-05-25

docx  (15.9 KB)