Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA
Cavaller-Bellaubi, Maria, Hughes-Wilson, Wills, Kubinová, Šárka, Van de Casteele, Marc, Van Lente, Evert Jan, Degortes, Emanuele, Pontén, Johan, Eichler, Hans-Georg, Le Cam, Yann, Boselli, Simone, Bucsics, Anna
Published in Orphanet journal of rare diseases (12.06.2023)
Published in Orphanet journal of rare diseases (12.06.2023)
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Journal Article
Drug repurposing for rare: progress and opportunities for the rare disease community
Jonker, Anneliene Hechtelt, O'Connor, Daniel, Cavaller-Bellaubi, Maria, Fetro, Christine, Gogou, Maria, 't Hoen, Peter A C, de Kort, Martin, Stone, Heather, Valentine, Nivedita, Pasmooij, Anna Maria Gerdina
Published in Frontiers in medicine (2024)
Published in Frontiers in medicine (2024)
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Journal Article
Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action
Cavaller-Bellaubi, Maria, Faulkner, Stuart D., Teixeira, Bryan, Boudes, Mathieu, Molero, Eva, Brooke, Nicholas, McKeaveney, Laura, Southerton, Jeffrey, Vicente, Maria José, Bertelsen, Neil, García-Burgos, Juan, Pirard, Vinciane, Reid, Kirsty, Ferrer, Elisa
Published in Therapeutic innovation & regulatory science (01.09.2021)
Published in Therapeutic innovation & regulatory science (01.09.2021)
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Journal Article
Access in the rare diseases landscape
Jonker, Anneliene H, Cavaller-Bellaubi, Maria, Nishimura, Yukiko, Pearce, David A
Published in The Lancet global health (01.10.2024)
Published in The Lancet global health (01.10.2024)
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Journal Article
Paediatric Inflammatory Bowel Disease: A Multi-Stakeholder Perspective to Improve Development of Drugs for Children and Adolescents
Croft, Nicholas M, de Ridder, Lissy, Griffiths, Anne M, Hyams, Jeffrey S, Ruemmele, Frank M, Turner, Dan, Cheng, Katharine, Lutsar, Irja, Greco, Marco, Gołębiewska, Zuzanna, Laumond, Floriane, Cavaller-Bellaubi, Maria, Elgreey, Adam, Altepeter, Tara A, Pallidis, Chrissi, Norga, Koen, Nelson, Robert, Crandall, Wallace, Vassal, Gilles
Published in Journal of Crohn's and colitis (18.03.2023)
Published in Journal of Crohn's and colitis (18.03.2023)
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Journal Article