Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases POSITION STATEMENT From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research Petra Kaufmann, Anne R. Pariser and Christopher Austin Orphanet Journal of Rare Diseases 2018, 13:196 | Published on: 6 November 2018 Full Text | PDF POSITION STATEMENT Recommendations for the design of small population clinical trials Simon Day, Anneliene Hechtelt Jonker, Lilian Pek Lian Lau, Ralf-Dieter Hilgers, Ilan Irony, Kristina Larsson, Kit CB Roes and Nigel Stallard Orphanet Journal of Rare Diseases 2018, 13:195 | Published on: 6 November 2018 Full Text | PDF RESEARCH The European challenges of funding orphan medicinal products 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 Orphanet Journal of Rare Diseases 2018, 13:184 | Published on: 6 November 2018 Full Text | PDF

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