Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multi-center disease monitoring program (DMP) by Giugliani, Roberto, Grant, Christina L., Scarpa, Maurizio, Sun, Angela, Wang, Raymond Y., Burton, Barbara K., Oussoren, Esmeralda, Durand, Consuelo, Martins, Esmeralda E., Chabrol, Brigitte, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence, Gonzalez-Meneses, Antonio

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Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis ty... by Malkus, Betsy C., Cohen, Jeanne M., Grant, Christina L., Scarpa, Maurizio, Sun, Angela, Wang, Raymond Y., Burton, Barbara K., Oussoren, Esmeralda, Martins, Esmeralda E., Durand, Consuelo, Chabrol, Brigitte, Ortiz, Lionel, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence, Giugliani, Roberto, Gonzalez-Meneses, Antonio

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Phenylketonuria in Portugal: Genotype–phenotype correlations using molecular, biochemical, and haplotypic analyses by Ferreira, Filipa, Azevedo, Luísa, Neiva, Raquel, Sousa, Carmen, Fonseca, Helena, Marcão, Ana, Rocha, Hugo, Carmona, Célia, Ramos, Sónia, Bandeira, Anabela, Martins, Esmeralda, Campos, Teresa, Rodrigues, Esmeralda, Garcia, Paula, Diogo, Luísa, Ferreira, Ana Cristina, Sequeira, Silvia, Silva, Francisco, Rodrigues, Luísa, Gaspar, Ana, Janeiro, Patrícia, Amorim, António, Vilarinho, Laura

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Continuous use of glycomacropeptide in the nutritional management of patients with phenylketonuria: a clinical perspective by Pena, Maria João, Pinto, Alex, de Almeida, Manuela Ferreira, de Sousa Barbosa, Catarina, Ramos, Paula Cristina, Rocha, Sara, Guimas, Arlindo, Ribeiro, Rosa, Martins, Esmeralda, Bandeira, Anabela, Dias, Cláudia Camila, MacDonald, Anita, Borges, Nuno, Rocha, Júlio César

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Lipid Antigen Presentation by CD1b and CD1d in Lysosomal Storage Disease Patients by Pereira, Catia S, Pérez-Cabezas, Begoña, Ribeiro, Helena, Maia, M Luz, Cardoso, M Teresa, Dias, Ana F, Azevedo, Olga, Ferreira, M Fatima, Garcia, Paula, Rodrigues, Esmeralda, Castro-Chaves, Paulo, Martins, Esmeralda, Aguiar, Patricio, Pineda, Mercè, Amraoui, Yasmina, Fecarotta, Simona, Leão-Teles, Elisa, Deng, Shenglou, Savage, Paul B, Macedo, M Fatima

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Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders—A successful strategy for clinical research of rare diseases by Posset, Roland, Garbade, Sven F., Boy, Nikolas, Burlina, Alberto B., Dionisi‐Vici, Carlo, Dobbelaere, Dries, Garcia‐Cazorla, Angeles, de Lonlay, Pascale, Teles, Elisa Leão, Vara, Roshni, Mew, Nicholas Ah, Batshaw, Mark L., Baumgartner, Matthias R., McCandless, Shawn E., Seminara, Jennifer, Summar, Marshall, Hoffmann, Georg F., Kölker, Stefan, Burgard, Peter, Berry, Susan A., Burrage, Lindsay, Coughlin, Curtis, Diaz, George A., Gallagher, Renata C., Gropman, Andrea, Harding, Cary O., Lee, Brendan, Le Mons, Cynthia, Lawrence Merritt, J., Nagamani, Sandesh C. S., Schulze, Andreas, Stricker, Tamar, Tuchman, Mendel, Waisbren, Susan, WeisfeldAdams, James, Wong, Derek, Yudkoff, Marc, Arnoux, JeanBaptiste, Bari, Ivo, Bosch, Annet M., Chabrol, Brigitte, Chakrapani, Anupam, CortèsSaladefont, Elisenda, Couce, Maria L., Eyskens, Francois, Laet, Corine, Meirleir, Linda, Freisinger, Peter, Gleich, Florian, Grünewald, Stephanie, Häberle, Johannes, Hwu, WuhLiang, Jalan, Anil, Karall, Daniela, Lindner, Martin, Lund, Allan M., Martinelli, Diego, Murphy, Elaine, Mühlhausen, Chris, Olivieri, Giorgia, Ottolenghi, Chris, Rodrigues, Esmeralda, Rubert, Laura, Sarajlija, Adrijan, Schiff, Manuel, Sokal, Etienne, SykutCegielska, Jolanta, Walter, John H., Williams, Monique, Zeman, Jiri

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The European reference network for metabolic diseases by Parenti, Giancarlo, Fecarotta, Simona, Alagia, Marianna, Attaianese, Federica, Verde, Alessandra, Tarallo, Antonietta, Gragnaniello, Vincenza, Ziagaki, Athanasia, Guimaraes, Maria Jose, Aguiar, Patricio, Hahn, Andreas, Azevedo, Olga, Donati, Maria Alice, Kiec-Wilk, Beata, Scarpa, Maurizio, van der Beek, Nadine A. M. E, Del Toro Riera, Mireja, Germain, Dominique P, Huidekoper, Hidde, van den Hout, Johanna M. P, van der Ploeg, Ans T, Baric, Ivo, Batzios, Spyros, Belmatoug, Nadia, Bordugo, Andrea, Bosch, Annet M, Brassier, Anais, Burlina, Alberto, Cassiman, David, Chabrol, Brigitte, Chronopoulou, Efstathia, Couce-Pico, Maria Luz, Darin, Niklas, Das, Anibh M, Debray, Francois G, Deegan, Patrick, de Abreu Freire Diogo Matos, Luisa M, De Las Heras Montero, Javier, Di Rocco, Maja, Dobbelaere, Dries, Eyskens, Francois, Ferreira, Ana, Gaspar, Ana M, Gasperini, Serena, López, Antonio González-Meneses, Grosso, Salvatore, Guffon-Fouilhoux, Nathalie, Hennermann, Julia, Hiwot, Tarekegn G, Jones, Simon, Kingma, Sandra, Komninaka, Veroniki, Martín-Hernández, Elena, Martins, Esmeralda, Miclea, Diana, Pfliegler, György, Rodrigues, Esmeralda, Rokicki, Dariusz, Roland, Dominique, Rutsch, Frank, Salviati, Alessandro, Tournev, Ivailo, Ullrich, Kurt, van Hasselt, Peter M, Vijay, Suresh, Weinhold, Natalie, Witters, Peter, Zeman, Jiri

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Feasibility of a multidisciplinary group videoconferencing approach for chronic low back pain: a randomized, open-label, controlled, pilot clinical trial (EN-FORMA) by Garreta-Catala, Iago, Planas-Balagué, Rosa, Abouzari, Reza, Carnaval, Thiago, Nolla, Joan M, Videla, Sebastián, Agulló-Ferré, José-Luis

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I-SPY COVID adaptive platform trial for COVID-19 acute respiratory failure: rationale, design and operations by Files, Daniel Clark, Calfee, Carolyn S, Asare, Adam L, Beitler, Jeremy R, Berger, Paul A, Burnham, Ellen L, Cimino, George, Coleman, Melissa H, Crippa, Alessio, Discacciati, Andrea, Gandotra, Sheetal, Ittner, Caroline A G, Jauregui, Alejandra, Khan, Kashif T, Koff, Jonathan L, Levitt, Joe, Lu, Ruixiao, McKeehan, Jeffrey D, Meyer, Nuala J, Russell, Derek W, Thomas, Karl W, Esserman, Laura J, Liu, Kathleen D, Liu, Kathleen D, Matthay, Michael A, Beitler, Jeremy R, Asare, Adam L, Yen, Albert F, Jauregui, Alejandra, Crippa, Alessio, Rosen, Amanda, Dzierba, Amy L, Discacciati, Andrea, Haczku, Angela, Barker, Anna D, Weisman, Ariel R, Daniel, Brian M, Morrissey, Brian M, Jones, Chayse, Files, Daniel C, Russell, Derek W, Ng, Diana, Burnham, Ellen L, Youssef, Fady A, Chaparro-Rojas, Fredy, Barot, Harsh V, Su, Heny, Sutter, Jacqueline B, Dodin, Jamal, Lee, Jerry S, Kazianis, John, Koff, Jonathan L, Detelich, Joshua F, Lang, Julie E, Thomas, Karl W, Gosek, Katarzyna, Nugent, Katherine L, Yee, Kimberly, Rodrigues, Laura G, Orr, Lindsey A, Rosario-Remigio, Lissette, Huerta, Luis E, Reyes, Maria B, Eklund, Martin, LaRose, Mary, Adelman, Max W, Juarez, Maya M, Jung, Michelle, Cobb, Nathan K, Aggarwal, Neil, Berger, Paul A, Saban, Paul L, Marshall, Peter S, Robinson, Philiip A, Yang, Philip, Madahar, Purnema, Nair, Rahul, Wunderink, Richard G, Wahab, Romina, Lupu, Roxana A, Lu, Ruixiao, Kumar, Santhi I, Auld, Sara C, Fields, Scott, Gandotra, Sheetal, Whealon, Spencer, Albertson, Timothy E, Obermiller, Timothy F, Oliver, Tony, Schicchi, John, Sarafian, Farjad, Nguyen, Julie, Blevins, Daniel, Melendrez, Marylee, Tzehaie, Hiwet, Amosu, Omowunmi, Levitt, Joe, Peterfreund, Gregory, Landreth, Leigha, Parks, Lisa

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Utility of Gene Panels for the Diagnosis of Inborn Errors of Metabolism in a Metabolic Reference Center by Barbosa-Gouveia, Sofia, Vázquez-Mosquera, María E, González-Vioque, Emiliano, Álvarez, José V, Chans, Roi, Laranjeira, Francisco, Martins, Esmeralda, Ferreira, Ana Cristina, Avila-Alvarez, Alejandro, Couce, María L

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Systematic review of guidelines for internal validity in the design, conduct and analysis of preclinical biomedical experiments involving laboratory animals by Vollert, Jan, Schenker, Esther, Macleod, Malcolm, Bespalov, Anton, Wuerbel, Hanno, Michel, Martin, Dirnagl, Ulrich, Potschka, Heidrun, Waldron, Ann-Marie, Wever, Kimberley, Steckler, Thomas, van de Casteele, Tom, Altevogt, Bruce, Sil, Annesha, Rice, Andrew S C

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Research activity and capability in the European reference network MetabERN by Heard, Jean-Michel, Bellettato, Cinzia, van Lingen, Corine, Scarpa, Maurizio

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Correction to: Age at disease onset and peak ammonium level rather than interventional variables predict the neurological outcome in urea cycle disorders by Posset, Roland, Garcia-Cazorla, Angeles, Valayannopoulos, Vassili, Leão Teles, Elisa, Dionisi-Vici, Carlo, Brassier, Anaïs, Burlina, Alberto B., Burgard, Peter, Cortès-Saladelafont, Elisenda, Dobbelaere, Dries, Couce, Maria L., Sykut-Cegielska, Jolanta, Häberle, Johannes, Lund, Allan M., Chakrapani, Anupam, Schiff, Manuel, Walter, John H., Zeman, Jiri, Vara, Roshni, Kölker, Stefan

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Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-a successful strategy for clinical research of rare diseases by Posset, Roland, Garbade, Sven F, Boy, Nikolas, Burlina, Alberto B, Dionisi-Vici, Carlo, Dobbelaere, Dries, Garcia-Cazorla, Angeles, de Lonlay, Pascale, Teles, Elisa Leão, Vara, Roshni, Ah Mew, Nicholas, Batshaw, Mark L, Baumgartner, Matthias R, McCandless, Shawn, Seminara, Jennifer, Summar, Marshall, Hoffmann, Georg F, Kölker, Stefan, Burgard, Peter

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Newborn screening for medium-chain acyl-CoA dehydrogenase deficiency: regional experience and high incidence of carnitine deficiency by Couce, Maria Luz, Sánchez-Pintos, Paula, Diogo, Luisa, Leão-Teles, Elisa, Martins, Esmeralda, Santos, Helena, Bueno, Maria Amor, Delgado-Pecellín, Carmen, Castiñeiras, Daisy E, Cocho, José A, García-Villoria, Judit, Ribes, Antonia, Fraga, José M, Rocha, Hugo

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Infantile Refsum Disease: Influence of Dietary Treatment on Plasma Phytanic Acid Levels by Sá, Maria João Nabais, Rocha, Júlio C., Almeida, Manuela F., Carmona, Carla, Martins, Esmeralda, Miranda, Vasco, Coutinho, Miguel, Ferreira, Rita, Pacheco, Sara, Laranjeira, Francisco, Ribeiro, Isaura, Fortuna, Ana Maria, Lacerda, Lúcia

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Maple syrup disease presenting as paroxysmal dystonia by Temudo, Teresa, Martins, Esmeralda, Poças, Fátima, Cruz, Romeu, Vilarinho, Laura

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Atitudes dos enfermeiros frente à morte no contexto hospitalar: diferenciação por unidades de cuidados by Cardoso, Maria Filomena Passos Teixeira, Martins, Maria Manuela Ferreira Pereira da Silva, Ribeiro, Olga Maria Pimenta Lopes, Fonseca, Esmeralda Faria

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La pandemia por COVID-19 y las actitudes de los enfermeros frente a la muerte by Maria Filomena Passos Teixeira Cardoso, Maria Manuela Ferreira Pereira da Silva Martins, Letícia de Lima Trindade, Olga Maria Pimenta Lopes Ribeiro, Esmeralda Faria Fonseca

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A pandemia por COVID-19 e as atitudes dos enfermeiros frente à morte by Maria Filomena Passos Teixeira Cardoso, Maria Manuela Ferreira Pereira da Silva Martins, Letícia de Lima Trindade, Olga Maria Pimenta Lopes Ribeiro, Esmeralda Faria Fonseca

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