A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial-Reference-Cited by-同舟云学术

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

Published:2018-05-10 Issue:1 Volume:19 Page:
ISSN:1745-6215
Container-title:Trials
language:en
Short-container-title:Trials

Author:

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

Funder

National Institute of Neurological Disorders and Stroke

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Medicine (miscellaneous)

Link

http://link.springer.com/content/pdf/10.1186/s13063-018-2645-0.pdf

Reference34 articles.

1. International rare disease research consortium. Policies and guidelines. 2013. http://www.irdirc.org/wp-content/uploads/2017/10/IRDiRC_policies_24MayApr2013.pdf . Accessed 8 May 2018.

2. International rare disease research consortium. Visions and goals. http://www.irdirc.org/about-us/vision-goals/ . Accessed May 2018.

3. Griggs RC, et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab. 2009;96:20–6.

4. Atal I, Trinquart L, Porcher R, Ravaud P. Differential globalization of industry- and non-industry-sponsored clinical trials. PLoS One. 2015;10:e0145122.

5. Minisman G, et al. Implementing clinical trials on an international platform: challenges and perspectives. J Neurol Sci. 2012; https://doi.org/10.1016/j.jns.2011.10.004 .

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