Patient selection considerations for AADC deficiency gene therapy by Agathe Roubertie, Irina Anselm, Bruria Ben‐Zeev, Wuh‐Liang Hwu, Ashutosh Kumar, Berrin Monteleone, Shin‐ichi Muramatsu, Vincenzo Leuzzi, Salvador Ibáñez, Scellig Stone, Phillip L. Pearl

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iPSC‐based modeling of THD recapitulates disease phenotypes and reveals neuronal malformation by Alba Tristán‐Noguero, Irene Fernández‐Carasa, Carles Calatayud, Cristina Bermejo‐Casadesús, Meritxell Pons‐Espinal, Arianna Colini Baldeschi, Leticia Campa, Francesc Artigas, Analia Bortolozzi, Rosario Domingo‐Jiménez, Salvador Ibáñez, Mercè Pineda, Rafael Artuch, Ángel Raya, Àngels García‐Cazorla, Antonella Consiglio

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A Multi-Target Pharmacological Correction of a Lipoyltransferase <i>LIPT1</i> Gene Mutation in Patient-Derived Cellular Models by David Gómez-Fernández, Ana Romero-González, Juan M. Suárez-Rivero, Paula Cilleros-Holgado, Mónica Álvarez-Córdoba, Rocío Piñero-Pérez, José Manuel Romero-Domínguez, Diana Reche-López, Alejandra López-Cabrera, Salvador Ibáñez-Mico, Marta Castro de Oliveira, Andrés Rodríguez-Sacristán, Susana González-Granero, José Manuel García-Verdugo, José A. Sánchez-Alcázar

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Impact of COVID19 pandemic on patients with rare diseases in Spain, with a special focus on inherited metabolic diseases by M. Mar Rovira-Remisa, Mónica Moreira, Paula Sol Ventura, Pablo Gonzalez-Alvarez, Núria Mestres, Fredzzia Graterol Torres, Clara Joaquín, Agustí Rodríguez-Palmero Seuma, Maria del Mar Martínez-Colls, Ana Roche, Salvador Ibáñez-Micó, Eduardo López-Laso, María Jesús Méndez-Hernández, Marta Murillo, Laura Monlleó-Neila, Elena Maqueda-Castellote, Mireia del Toro Riera, Ana Felipe-Rucián, Maria Giralt-López, Elisenda Cortès-Saladelafont

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Availability and use of rapid diagnostic tests for the management of acute childhood infections in Europe: A cross-sectional survey of paediatricians. by Juan Emmanuel Dewez, Lucy Pembrey, Ruud G Nijman, Stefano Del Torso, Zachi Grossman, Adamos Hadjipanayis, Diego Van Esso, Emma Lim, Marieke Emonts, James Burns, Christèle Gras-LeGuen, Daniela Kohlfuerst, Hans Jürgen Dornbusch, Karen Brengel-Pesce, Francois Mallet, Ulrich von Both, Maria Tsolia, Irini Eleftheriou, Dace Zavadska, Ronald de Groot, Michiel van der Flier, Henriëtte Moll, Nienke Hagedoorn, Dorine Borensztajn, Rianne Oostenbrink, Taco Kuijpers, Marko Pokorn, Katarina Vincek, Federico Martinón-Torres, Irene Rivero, Philipp Agyeman, Enitan D Carrol, Stéphane Paulus, Aubrey Cunnington, Jethro Herberg, Michael Levin, Aida Mujkić, Karin Geitmann, Liviana Da Dalt, Arūnas Valiulis, Risto Lapatto, Garyfallia Syridou, Péter Altorjai, Paul Torpiano, Ketil Størdal, Károly Illy, Artur Mazur, Mateja Vintar Spreitzer, Joana Rios, Corinne Wyder, Ivanna Romankevych, Romain Basmaci, Salvador Ibanez-Mico, Shunmay Yeung

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Developmental outcome of electroencephalographic findings in SYNGAP1 encephalopathy by Juliana Ribeiro-Constante, Alba Tristán-Noguero, Alba Tristán-Noguero, Fernando Francisco Martínez Calvo, Salvador Ibañez-Mico, José Luis Peña Segura, José Miguel Ramos-Fernández, María del Carmen Moyano Chicano, Rafael Camino León, Víctor Soto Insuga, Elena González Alguacil, Carlos Valera Dávila, Alberto Fernández-Jaén, Laura Plans, Ana Camacho, Nuria Visa-Reñé, María del Pilar Martin-Tamayo Blázquez, Fernando Paredes-Carmona, Itxaso Marti-Carrera, Aránzazu Hernández-Fabián, Meritxell Tomas Davi, Merce Casadesus Sanchez, Laura Cuesta Herraiz, Patricia Fuentes Pita, Teresa Bermejo Gonzalez, Mar O'Callaghan, Federico Felipe Iglesias Santa Polonia, María Rosario Cazorla, María Teresa Ferrando Lucas, Antonio González-Meneses, Júlia Sala-Coromina, Alfons Macaya, Amaia Lasa-Aranzasti, Anna Ma Cueto-González, Francisca Valera Párraga, Jaume Campistol Plana, Mercedes Serrano, Xenia Alonso, Diego Del Castillo-Berges, Marc Schwartz-Palleja, Marc Schwartz-Palleja, Marc Schwartz-Palleja, Sofía Illescas, Alia Ramírez Camacho, Oscar Sans Capdevila, Angeles García-Cazorla, Àlex Bayés, Itziar Alonso-Colmenero

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