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1
CD19 CAR T cell product and disease attributes predict leukemia remission durability
CD19 CAR T cell product and disease attributes predict leukemia remission durability
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CD19 CAR T cell product and disease attributes predict leukemia remission durability

Finney, Olivia C ; Brakke, Hannah M ; Rawlings-Rhea, Stephanie ; Hicks, Roxana ; Doolittle, Danielle ; Lopez, Marisa ; Futrell, Robert B ; Orentas, Rimas J ; Li, Daniel ; Gardner, Rebecca A ; Jensen, Michael C

The Journal of clinical investigation, 2019-05, Vol.129 (5), p.2123-2132 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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2
Dual CD19 and CD123 targeting prevents antigen-loss relapses after CD19-directed immunotherapies
Dual CD19 and CD123 targeting prevents antigen-loss relapses after CD19-directed immunotherapies
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Dual CD19 and CD123 targeting prevents antigen-loss relapses after CD19-directed immunotherapies

Ruella, Marco ; Barrett, David M ; Kenderian, Saad S ; Shestova, Olga ; Hofmann, Ted J ; Perazzelli, Jessica ; Klichinsky, Michael ; Aikawa, Vania ; Nazimuddin, Farzana ; Kozlowski, Miroslaw ; Scholler, John ; Lacey, Simon F ; Melenhorst, Jan J ; Morrissette, Jennifer J D ; Christian, David A ; Hunter, Christopher A ; Kalos, Michael ; Porter, David L ; June, Carl H ; Grupp, Stephan A ; Gill, Saar

The Journal of clinical investigation, 2016-10, Vol.126 (10), p.3814-3826 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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3
A roadmap to using historical controls in clinical trials - by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG)
A roadmap to using historical controls in clinical trials - by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG)
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A roadmap to using historical controls in clinical trials - by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG)

Ghadessi, Mercedeh ; Tang, Rui ; Zhou, Joey ; Liu, Rong ; Wang, Chenkun ; Toyoizumi, Kiichiro ; Mei, Chaoqun ; Zhang, Lixia ; Deng, C Q ; Beckman, Robert A

Orphanet journal of rare diseases, 2020-03, Vol.15 (1), p.69-69, Article 69 [Periódico revisado por pares]

England: BioMed Central

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4
DNA methylation heterogeneity defines a disease spectrum in Ewing sarcoma
DNA methylation heterogeneity defines a disease spectrum in Ewing sarcoma
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DNA methylation heterogeneity defines a disease spectrum in Ewing sarcoma

Sheffield, Nathan C ; Pierron, Gaelle ; Klughammer, Johanna ; Datlinger, Paul ; Schönegger, Andreas ; Schuster, Michael ; Hadler, Johanna ; Surdez, Didier ; Guillemot, Delphine ; Lapouble, Eve ; Freneaux, Paul ; Champigneulle, Jacqueline ; Bouvier, Raymonde ; Walder, Diana ; Ambros, Ingeborg M ; Hutter, Caroline ; Sorz, Eva ; Amaral, Ana T ; de Álava, Enrique ; Schallmoser, Katharina ; Strunk, Dirk ; Rinner, Beate ; Liegl-Atzwanger, Bernadette ; Huppertz, Berthold ; Leithner, Andreas ; de Pinieux, Gonzague ; Terrier, Philippe ; Laurence, Valérie ; Michon, Jean ; Ladenstein, Ruth ; Holter, Wolfgang ; Windhager, Reinhard ; Dirksen, Uta ; Ambros, Peter F ; Delattre, Olivier ; Kovar, Heinrich ; Bock, Christoph ; Tomazou, Eleni M

Nature medicine, 2017-03, Vol.23 (3), p.386-395 [Periódico revisado por pares]

United States: Nature Publishing Group

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5
Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
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Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis

Coratti, Giorgia ; Cutrona, Costanza ; Pera, Maria Carmela ; Bovis, Francesca ; Ponzano, Marta ; Chieppa, Fabrizia ; Antonaci, Laura ; Sansone, Valeria ; Finkel, Richard ; Pane, Marika ; Mercuri, Eugenio

Orphanet journal of rare diseases, 2021-10, Vol.16 (1), p.1-430, Article 430 [Periódico revisado por pares]

London: BioMed Central Ltd

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6
Tumor Antigen and Receptor Densities Regulate Efficacy of a Chimeric Antigen Receptor Targeting Anaplastic Lymphoma Kinase
Tumor Antigen and Receptor Densities Regulate Efficacy of a Chimeric Antigen Receptor Targeting Anaplastic Lymphoma Kinase
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Tumor Antigen and Receptor Densities Regulate Efficacy of a Chimeric Antigen Receptor Targeting Anaplastic Lymphoma Kinase

Walker, Alec J. ; Majzner, Robbie G. ; Zhang, Ling ; Wanhainen, Kelsey ; Long, Adrienne H. ; Nguyen, Sang M. ; Lopomo, Paola ; Vigny, Marc ; Fry, Terry J. ; Orentas, Rimas J. ; Mackall, Crystal L.

Molecular therapy, 2017-09, Vol.25 (9), p.2189-2201 [Periódico revisado por pares]

United States: Elsevier Inc

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7
Vaccine confidence among parents: Large scale study in eighteen European countries
Vaccine confidence among parents: Large scale study in eighteen European countries
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Vaccine confidence among parents: Large scale study in eighteen European countries

Hadjipanayis, Adamos ; van Esso, Diego ; del Torso, Stefano ; Dornbusch, Hans Jürgen ; Michailidou, Kyriaki ; Minicuci, Nadia ; Pancheva, Ruzha ; Mujkic, Aida ; Geitmann, Karin ; Syridou, Garyfallia ; Altorjai, Peter ; Pasinato, Angela ; Valiulis, Arunas ; Soler, Paul ; Cirstea, Olga ; Illy, Károly ; Mollema, Liesbeth ; Mazur, Artur ; Neves, Ana ; Zavrsnik, Jernej ; Lapii, Fedir ; Efstathiou, Elisavet ; Kamphuis, Mascha ; Grossman, Zachi

Vaccine, 2020-02, Vol.38 (6), p.1505-1512 [Periódico revisado por pares]

Netherlands: Elsevier Ltd

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8
ERNICA guidelines for the management of rectosigmoid Hirschsprung's disease
ERNICA guidelines for the management of rectosigmoid Hirschsprung's disease
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ERNICA guidelines for the management of rectosigmoid Hirschsprung's disease

Kyrklund, Kristiina ; Sloots, Cornelius E J ; de Blaauw, Ivo ; Bjørnland, Kristin ; Rolle, Udo ; Cavalieri, Duccio ; Francalanci, Paola ; Fusaro, Fabio ; Lemli, Annette ; Schwarzer, Nicole ; Fascetti-Leon, Francesco ; Thapar, Nikhil ; Johansen, Lars Søndergaard ; Berrebi, Dominique ; Hugot, Jean-Pierre ; Crétolle, Célia ; Brooks, Alice S ; Hofstra, Robert M ; Wester, Tomas ; Pakarinen, Mikko P

Orphanet journal of rare diseases, 2020-06, Vol.15 (1), p.164-16, Article 164 [Periódico revisado por pares]

England: BioMed Central Ltd

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9
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
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Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study

Audic, Frédérique ; de la Banda, Marta Gomez Garcia ; Bernoux, Delphine ; Ramirez-Garcia, Paola ; Durigneux, Julien ; Barnerias, Christine ; Isapof, Arnaud ; Cuisset, Jean-Marie ; Cances, Claude ; Richelme, Christian ; Vuillerot, Carole ; Laugel, Vincent ; Ropars, Juliette ; Altuzarra, Cécilia ; Espil-Taris, Caroline ; Walther-Louvier, Ulrike ; Sabouraud, Pascal ; Chouchane, Mondher ; Vanhulle, Catherine ; Trommsdorff, Valérie ; Pervillé, Anne ; Testard, Hervé ; Lagrue, Emmanuelle ; Sarret, Catherine ; Avice, Anne-Laude ; Beze-Beyrie, Pierre ; Pauly, Vanessa ; Quijano-Roy, Susana ; Chabrol, Brigitte ; Desguerre, Isabelle

Orphanet journal of rare diseases, 2020-06, Vol.15 (1), p.1-148, Article 148 [Periódico revisado por pares]

London: BioMed Central Ltd

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10
Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium
Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium
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Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium

Vandeborne, Liese ; van Overbeeke, Eline ; Dooms, Marc ; De Beleyr, Birgit ; Huys, Isabelle

Orphanet journal of rare diseases, 2019-05, Vol.14 (1), p.99-11, Article 99 [Periódico revisado por pares]

England: BioMed Central Ltd

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