Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database von Nguengang Wakap, Stéphanie, Lambert, Deborah M, Olry, Annie, Rodwell, Charlotte, Gueydan, Charlotte, Lanneau, Valérie, Murphy, Daniel, Le Cam, Yann, Rath, Ana

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Progress in Rare Diseases Research 2010–2016: An IRDiRC Perspective von 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, Cam, Yann, Hivert, Virginie, Austin, Christopher P.

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Reply to E. Vicente et al von Rath, Ana, Lambert, Deborah M., Olry, Annie, Rodwell, Charlotte, Le Cam, Yann

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Our greatest untapped resource: our patients von Bolz-Johnson, Matt, Kenny, Tom, Le Cam, Yann, Hernando, Ines

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Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Prop... von Aartsma-Rus, Annemieke, Dooms, Marc, Le Cam, Yann

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Medicines Adaptive Pathways to Patients: Why, When, and How to Engage? von Eichler, Hans‐Georg, Bedlington, Nicola, Boudes, Mathieu, Bouvy, Jacoline C., Broekmans, André W., Cerreta, Francesca, Faulkner, Stuart D., Forda, Susan R., Joos, Angelika, Cam, Yann, Mayer, Mark H., Pirard, Vinciane, Corriol‐Rohou, Solange

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Towards Achieving Equity and Innovation in Newborn Screening across Europe von Sikonja, Jaka, Groselj, Urh, Scarpa, Maurizio, la Marca, Giancarlo, Cheillan, David, Kölker, Stefan, Zetterström, Rolf H, Kožich, Viktor, Le Cam, Yann, Gumus, Gulcin, Bottarelli, Valentina, van der Burg, Mirjam, Dekkers, Eugenie, Battelino, Tadej, Prevot, Johan, Schielen, Peter C J I, Bonham, James R

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The evolution of adaptiveness: balancing speed and evidence von Eichler, Hans-Georg, Barker, Richard, Bedlington, Nicola, Bouvy, Jacoline C., Broekmans, André W., Bucsics, Anna, Cerreta, Francesca, Corriol-Rohou, Solange, Granados, Alicia, Le Cam, Yann, Schuurman, Ad

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Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) von Annemans, Lieven, Aymé, Ségolène, Le Cam, Yann, Facey, Karen, Gunther, Penilla, Nicod, Elena, Reni, Michele, Roux, Jean-Louis, Schlander, Michael, Taylor, David, Tomino, Carlo, Torrent-Farnell, Josep, Upadhyaya, Sheela, Hutchings, Adam, Le Dez, Lugdivine

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The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact von Lochmüller, Hanns, Torrent I Farnell, Josep, Le Cam, Yann, Jonker, Anneliene H, Lau, Lilian Pl, Baynam, Gareth, Kaufmann, Petra, Dawkins, Hugh Js, Lasko, Paul, Austin, Christopher P, Boycott, Kym M

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The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference Networks von Evangelista, Teresinha, Hedley, Victoria, Atalaia, Antonio, Johnson, Matt, Lynn, Stephen, Le Cam, Yann, Bushby, Kate

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Operational description of rare diseases: a reference to improve the recognition and visibility of rare diseases von Wang, Chiuhui Mary, Whiting, Amy Heagle, Rath, Ana, Anido, Roberta, Ardigò, Diego, Baynam, Gareth, Dawkins, Hugh, Hamosh, Ada, Le Cam, Yann, Malherbe, Helen, Molster, Caron M, Monaco, Lucia, Padilla, Carmencita D, Pariser, Anne R, Robinson, Peter N, Rodwell, Charlotte, Schaefer, Franz, Weber, Stefanie, Macchia, Flaminia

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Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA von 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

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Strengthening health systems for access to gene therapy in rare genetic disorders von Bhatia, Sonal, Le Cam, Yann, Carrion, Juan, Diamond, Lauren, Fennessy, Paul, Gassman, Safiyya, Gutzwiller, Felix, Kagan, Stephen, Pankevich, Diana, Young Maloney, Jennifer, Mahadev, Nitin, Schulz, Martin, Wong-Rieger, Durhane, Morgese, Paolo

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Interview: Yann Le Cam von Le Cam, Yann

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The added value of centres of expertise for rare disease patients in Europe von Kole, Anna A, le Cam, Yann

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Time to diagnosis and determinants of diagnostic delays of people living with a rare disease: results of a Rare Barometer retrospective patient survey von Faye, Fatoumata, Crocione, Claudia, Anido de Peña, Roberta, Bellagambi, Simona, Escati Peñaloza, Luciana, Hunter, Amy, Jensen, Lene, Oosterwijk, Cor, Schoeters, Eva, de Vicente, Daniel, Faivre, Laurence, Wilbur, Michael, Le Cam, Yann, Dubief, Jessie

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Progressive enlargement of filtrations with initial times von Jeanblanc, Monique, Le Cam, Yann

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Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA von 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

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L’action de la France en Europe et ce que l’Europe peut inspirer à la France, du point de vue des malades von Le Cam, Yann

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