A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned...
A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
About this item
Full title
Author / 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 and Guglieri, Michela
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
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 clinical trial regulations in those countries. Over the past years, there have been initiatives to facilitate the process of international study set-up, but the fruits o...
Alternative Titles
Full title
A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_59fc2ba5f36543fab1efe37a189c7b3f
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_59fc2ba5f36543fab1efe37a189c7b3f
Other Identifiers
ISSN
1745-6215
E-ISSN
1745-6215
DOI
10.1186/s13063-018-2645-0
