Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page



Orphanet Journal of Rare Diseases

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1. Content Type:Research

     |  9 November 2018

   Inhibitor clinical burden of disease: a comparative analysis of the CHESS data

   Authors:Abiola O. Oladapo, Mei Lu, Shaun Walsh, Jamie O’Hara and Teresa L. Kauf
2. Content Type:Research

     |  8 November 2018

   Mapping health care of rare diseases: the example of epidermolysis bullosa in Germany

   Authors:Antonia Reimer, Leena Bruckner-Tuderman and Hagen Ott
3. Content Type:Research

     |  6 November 2018

   The European challenges of funding orphan medicinal products

   Authors:Márta Szegedi, Tamás Zelei, Francis Arickx, Anna Bucsics, Emanuelle Cohn-Zanchetta, Jurij Fürst, Maria Kamusheva, Pawel Kawalec, Guenka Petrova, Juraj Slaby, Ewa Stawowczyk, Milan Vocelka, Ingrid Zechmeister-Koss, Zoltán Kaló and Mária Judit Molnár
4. Content Type:Position Statement

     |  6 November 2018

   Recommendations for the design of small population clinical trials

   Authors:Simon Day, Anneliene Hechtelt Jonker, Lilian Pek Lian Lau, Ralf-Dieter Hilgers, Ilan Irony, Kristina Larsson, Kit CB Roes and Nigel Stallard
5. Content Type:Position Statement

     |  6 November 2018

   From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research

   Authors:Petra Kaufmann, Anne R. Pariser and Christopher Austin

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