Worldwide collaboration for orphan drug designation von Mariz, Segundo, Reese, James H., Westermark, Kerstin, Greene, Lesley, Goto, Takahiro, Hoshino, Tatsuro, Llinares-Garcia, Jordi, Sepodes, Bruno

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Regulatory Standards in Orphan Medicinal Product Designation in the EU von Tsigkos, Stelios, Mariz, Segundo, Sheean, Maria Elzbieta, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta

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Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation von Mulder, Jorn, van Rossum, Tobias, Mariz, Segundo, Magrelli, Armando, de Boer, Anthonius, Pasmooij, Anna M. G., Stoyanova-Beninska, Violeta

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Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe von Farkas, Andreas M, Mariz, Segundo, Stoyanova-Beninska, Violeta, Celis, Patrick, Vamvakas, Spiros, Larsson, Kristina, Sepodes, Bruno

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Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective von Sheean, Maria E., Naumann-Winter, Frauke, Capovilla, Giuseppe, Kalland, Maria Elisabeth, Malikova, Eva, Mariz, Segundo, Matusevicius, Darius, Nistico, Robert, Schwarzer-Daum, Brigitte, Tsigkos, Stelios, Tzogani, Kyriaki, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta

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Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective von Tomeo, Francesca, Mariz, Segundo, Brunetta, Angelo Loris, Stoyanova‐Beninska, Violeta, Penttila, Karri, Magrelli, Armando

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Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe von Palomo, Gloria M., Pose-Boirazian, Tomas, Naumann-Winter, Frauke, Costa, Enrico, Duarte, Dinah M., Kalland, Maria E., Malikova, Eva, Matusevicius, Darius, Vitezic, Dinko, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta, Mariz, Segundo

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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation von Kalland, Maria Elisabeth, Pose-Boirazian, Tomas, Palomo, Gloria Maria, Naumann-Winter, Frauke, Costa, Enrico, Matusevicius, Darius, Duarte, Dinah M., Malikova, Eva, Vitezic, Dinko, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta, Mariz, Segundo

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Challenges in conducting clinical trials in nephrology: conclusions from a Kidney Disease—Improving Global Outcomes (KDIGO) Controversies Conference von Baigent, Colin, Herrington, William G., Coresh, Josef, Landray, Martin J., Levin, Adeera, Perkovic, Vlado, Pfeffer, Marc A., Rossing, Peter, Walsh, Michael, Wanner, Christoph, Wheeler, David C., Winkelmayer, Wolfgang C., McMurray, John J.V., Abu-Alfa, Ali, Archdeacon, Patrick, Block, Geoffrey A., Caskey, Fergus J., Cheung, Alfred K., Cooper, Bruce, Craig, Jonathan C., Dember, Laura M., Eknoyan, Garabed, Gansevoort, Ron T., Gill, John S., Gillespie, Barbara, Greene, Tom, Harris, David C., Haynes, Richard, Hemmelgarn, Brenda R., Herzog, Charles A., Hiemstra, Thomas F., Inker, Lesley A., Jardine, Meg J., Jha, Vivekanand, Jiang, Lixin, Johansen, Kirsten L., Kewalramani, Reshma, Lambers Heerspink, Hiddo J., Lefkowitz, Martin, Lok, Charmaine E., Loud, Fiona, Mačiulaitis, Romaldas, Maddux, Dugan W., Maddux, Franklin W., Madero, Magdalena, Mariz, Segundo, Mauer, Michael, Nally, Joseph V., Nangaku, Masaomi, Okpechi, Ikechi G., Parfrey, Patrick S., Pecoits-Filho, Roberto, Pereira, Brian J.G., Rocco, Michael V., Rossignol, Patrick, Schaefer, Franz, Tentori, Francesca, Thompson, Aliza, Tonelli, Marcello, Tong, Allison, Toto, Robert D., Tuttle, Katherine R., Vetter, Thorsten, Moon Wang, Angela Yee, Zannad, Faiez

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The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA committee for orphan medicinal products von Palomo, Gloria M., Pose-Boirazian, Thomas, Naumann-Winter, Frauke, Costa, Enrico, Duarte, Dinah M., Kalland, Maria E., Malikova, Eva, Matusevicius, Darius, Vitezic, Dinko, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta, Mariz, Segundo

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The orphan drug pipeline in Europe von Morel, Thomas, Lhoir, André, Picavet, Eline, Mariz, Segundo, Sepodes, Bruno, Llinares, Jordi, Cassiman, David

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Regulatory watch: The orphan drug pipeline in Europe von Morel, Thomas, Lhoir, André, Picavet, Eline, Mariz, Segundo, Sepodes, Bruno, Llinares, Jordi, Cassiman, David

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Establishing rarity in the context of orphan medicinal product designation in the European Union von Tsigkos, Stelios, Hofer, Matthias Philipp, Sheean, Maria Elzbieta, Mariz, Segundo, Larsson, Kristina, Naumann-Winter, Frauke, Fregonese, Laura, Sepodes, Bruno

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The EMA Assessment of Asciminib for the Treatment of Adult Patients With Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic Phase Who Were Previously Treated With... von Tesileanu, C Mircea S, Michaleas, Sotirios, Gonzalo Ruiz, Rocio, Mariz, Segundo, Fabriek, Babs O, van Hennik, Paula B, Dedorath, Jutta, Dekic, Bruna, Unkrig, Christoph, Brandt, Andreas, Koenig, Janet, Enzmann, Harald, Delgado, Julio, Pignatti, Francesco

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Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation von Mulder, Jorn, van Rossum, Tobias, Mariz, Segundo, Magrelli, Armando, de Boer, Anthonius, Pasmooij, Anna M.G, Stoyanova-Beninska, Violeta

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Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation von Mulder, Jorn, van Rossum, Tobias, Mariz, Segundo, Magrelli, Armando, de Boer, Anthonius, Pasmooij, Anna M.G, Stoyanova-Beninska, Violeta

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Common Elements in Rare Kidney Diseases: Conclusions from a Kidney Disease: Improving Global Outcomes (KDIGO) Controversies Conference von Aymé, Ségolène, Bockenhauer, Detlef, Day, Simon, Devuyst, Olivier, Guay-Woodford, Lisa M., Ingelfinger, Julie R., Klein, Jon B., Knoers, Nine V.A.M., Perrone, Ronald D., Roberts, Julia, Schaefer, Franz, Torres, Vicente E., Cheung, Michael, Wheeler, David C., Winkelmayer, Wolfgang C., Angelis, Aris, Antignac, Corinne, Bae, Kyongtae, Bergmann, Carsten, Bleyer, Anthony J., Bos, Marjolein, Budde, Klemens, Bull, Katherine, Chauveau, Dominique, Cnaan, Avital, Cornel, Martina, Cosyns, Etienne, de la Fosse, Jane, Ding, Jie, Gear, Susie, Goodship, Timothy H.J., Goodyer, Paul, Gross, Oliver, Harr, Nicole, Harris, Peter C., Harris, Tess, Höfele, Julia, Hogan, Marie C., Hoorn, Ewout, Horie, Shigeo, Kashtan, Clifford E., Kerecuk, Larissa, Kleta, Robert, Konrad, Martin, Langman, Craig B., Mariz, Segundo, McKerracher, Gayle, Nieuwenhoven, Annet, Odland, Dwight, Olinger, Eric, Ortiz, Alberto, Pei, York, Pirson, Yves, Rayner, Brian L., Remuzzi, Giuseppe, Renault, Daniel, Salomon, Rémi, Servais, Aude, Smith, Richard J., Soliman, Neveen A., Stengel, Bénédicte, Storm, Marjolein, Torra, Roser, van't Hoff, William, Vargas-Poussou, Rosa, Vroom, Elizabeth, Wanner, Christoph, Yap, Hui-Kim

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The orphan drug pipeline in Europe von Morel, Thomas, Lhoir, André, Picavet, Eline, Mariz, Segundo, Sepodes, Bruno, Llinares, Jordi, Cassiman, David

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Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union von Polsinelli, Benedetta, Tsigkos, Stelios, Naumann-Winter, Frauke, Mariz, Segundo, Sepodes, Bruno

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Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions von Sheean, Maria E., Stoyanova-Beninska, Violeta, Capovilla, Giuseppe, Duarte, Dinah, Hofer, Matthias P., Hoffmann, Michel, Magrelli, Armando, Mariz, Segundo, Tsigkos, Stelios, Shaili, Evyenia, Polsinelli, Benedetta, Ricciardi, Mario, Bonelli, Milton, Balabanov, Pavel, Larsson, Kristina, Sepodes, Bruno

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