New functionalities in Orphanet for orphan drugs, R&D and marketing authorisations to better serve the rare diseases community

Por Hivert, Virginie, Martin, Natalia, Hanauer, Marc, Aymé, Ségolène
Publicado em 2010

Rare diseases research in Europe: an overview based on data from the Orphanet database

Por Martin, Natalia, Doulet, Nicolas, Hivert, Virginie, Aymé, Ségolène
Publicado em 2010

The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining...

Por Southall, Noel T, Natarajan, Madhusudan, Lau, Lilian Pek Lian, Jonker, Anneliene Hechtelt, Deprez, Benoît, Guilliams, Tim, Hunter, Lawrence, Rademaker, Carin MA, Hivert, Virginie, Ardigò, Diego
Publicado no Orphanet J Rare Dis (2019)

Barriers to the conduct of randomised clinical trials within all disease areas

Por Djurisic, Snezana, Rath, Ana, Gaber, Sabrina, Garattini, Silvio, Bertele, Vittorio, Ngwabyt, Sandra-Nadia, Hivert, Virginie, Neugebauer, Edmund A. M., Laville, Martine, Hiesmayr, Michael, Demotes-Mainard, Jacques, Kubiak, Christine, Jakobsen, Janus C., Gluud, Christian
Publicado no Trials (2017)

Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review

Por Vat, Lidewij Eva, Finlay, Teresa, Jan Schuitmaker‐Warnaar, Tjerk, Fahy, Nick, Robinson, Paul, Boudes, Mathieu, Diaz, Ana, Ferrer, Elisa, Hivert, Virginie, Purman, Gabor, Kürzinger, Marie‐Laure, Kroes, Robert A., Hey, Claudia, Broerse, Jacqueline E.W.
Publicado no Health Expect (2019)

Progress in Rare Diseases Research 2010–2016: An IRDiRC Perspective

Por Dawkins, Hugh J.S., Draghia‐Akli, Ruxandra, Lasko, Paul, Lau, Lilian P.L., Jonker, Anneliene H., Cutillo, Christine M., Rath, Ana, Boycott, Kym M., Baynam, Gareth, Lochmüller, Hanns, Kaufmann, Petra, Le Cam, Yann, Hivert, Virginie, Austin, Christopher P.
Publicado no Clin Transl Sci (2017)

A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overco...

Por Rath, Ana, Salamon, Valérie, Peixoto, Sandra, Hivert, Virginie, Laville, Martine, Segrestin, Berenice, Neugebauer, Edmund A. M., Eikermann, Michaela, Bertele, Vittorio, Garattini, Silvio, Wetterslev, Jørn, Banzi, Rita, Jakobsen, Janus C., Djurisic, Snezana, Kubiak, Christine, Demotes-Mainard, Jacques, Gluud, Christian
Publicado no Trials (2017)

Future of Rare Diseases Research 2017–2027: An IRDiRC Perspective

Por Austin, Christopher P., Cutillo, Christine M., Lau, Lilian P.L., Jonker, Anneliene H., Rath, Ana, Julkowska, Daria, Thomson, David, Terry, Sharon F., de Montleau, Béatrice, Ardigò, Diego, Hivert, Virginie, Boycott, Kym M., Baynam, Gareth, Kaufmann, Petra, Taruscio, Domenica, Lochmüller, Hanns, Suematsu, Makoto, Incerti, Carlo, Draghia‐Akli, Ruxandra, Norstedt, Irene, Wang, Lu, Dawkins, Hugh J.S.
Publicado no Clin Transl Sci (2017)