Multidisciplinary care in haemoglobinopathies by Adriana Ceci, Laura Mangiarini, Fedele Bonifazi, Rossella Conte, on behalf of the HTA-THAL Multiregional Registry

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Target therapy for high-risk neuroblastoma treatment: integration of regulatory and scientific tools is needed by Adriana Ceci, Rosa Conte, Antonella Didio, Annalisa Landi, Lucia Ruggieri, Viviana Giannuzzi, Fedele Bonifazi

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Effects of the Paediatric Regulation funding on the development of off-patent medicines in children by Lucia Ruggieri, Silvia Torretta, Viviana Giannuzzi, Alessandra Natale, Mariagrazia Felisi, Adriana Ceci, Adriana Ceci, Fedele Bonifazi

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PB2515: A PILOT STUDY OF THE INTERNATIONAL HEMOGLOBINOPATHY RESEARCH NETWORK (INHERENT) by Petros Kountouris, Coralea Stephanou, Maxwell Nwegbu, Obiageli Nnodu, Morohuntodun Oni, Natasha Archer, Leon Tshilolo, Bin Alwi Zilfalil, Kyriaki Michailidou, Viviana Giannuzzi, Fedele Bonifazi, Marina Kleanthous

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Design and Implementation of a Sickle Cell Disease Electronic Registry in Resource Limited Setting in Nigeria—A Pilot Study by Muhammad Aminu Idris, Lucia Ruggieri, Hafsat Rufai Ahmad, Abdulaziz Hassan, Ismaila Nda Ibrahim, Faruk Jamil Adullahi, Sani Awwalu, Usman Nasiru, Fedele Bonifazi, Baba P. D. Inusa

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Mapping of Data-Sharing Repositories for Paediatric Clinical Research—A Rapid Review by Mariagrazia Felisi, Fedele Bonifazi, Maddalena Toma, Claudia Pansieri, Rebecca Leary, Victoria Hedley, Ronald Cornet, Giorgio Reggiardo, Annalisa Landi, Annunziata D’Ercole, Salma Malik, Sinéad Nally, Anando Sen, Avril Palmeri, Donato Bonifazi, Adriana Ceci

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Descriptive Analysis of Pediatric Studies Included in the European Union Post-Authorization Study Register from 2010 to 2023 by Annalisa Landi, Giorgio Reggiardo, Antonella Didio, Annunziata D’Ercole, Adriana Ceci, Grace Shalom Govere, Donato Bonifazi, Fedele Bonifazi, Salvatore Crisafulli, Gianluca Trifirò, Florentia Kaguelidou, Katja Marja Hakkarainen, Katarina Gvozdanović, Francesco Barone-Adesi, Andrealuna Ucciero, Mariagrazia Felisi

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Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities by Paul Dimitri, Valeria Pignataro, Mariangela Lupo, Donato Bonifazi, Maria Henke, Umberto M. Musazzi, Floris Ernst, Paola Minghetti, Davide F. Redaelli, Sophia G. Antimisiaris, Giovanni Migliaccio, Fedele Bonifazi, Luca Marciani, Aaron J. Courtenay, Nunzio Denora, Angela Lopedota

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Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia by Khaled M. Musallam, Angela Vitrano, Antonella Meloni, Sebastiano Addario Pollina, Mehran Karimi, Amal El-Beshlawy, Mahmoud Hajipour, Vito Di Marco, Saqib Hussain Ansari, Aldo Filosa, Paolo Ricchi, Adriana Ceci, Shahina Daar, Efthymia Vlachaki, Sylvia Titi Singer, Zaki A. Naserullah, Alessia Pepe, Salvatore Scondotto, Gabriella Dardanoni, Fedele Bonifazi, Vijay G. Sankaran, Elliott Vichinsky, Ali T. Taher, Aurelio Maggio

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Random Forest Clustering Identifies Three Subgroups of β-Thalassemia with Distinct Clinical Severity by Angela Vitrano, Khaled M. Musallam, Antonella Meloni, Sebastiano Addario Pollina, Mehran Karimi, Amal El-Beshlawy, Mahmoud Hajipour, Vito Di Marco, Saqib Hussain Ansari, Aldo Filosa, Paolo Ricchi, Adriana Ceci, Shahina Daar, Efthymia Vlachaki, Sylvia Titi Singer, Zaki A. Naserullah, Alessia Pepe, Salvatore Scondotto, Gabriella Dardanoni, Fedele Bonifazi, Vijay G. Sankaran, Elliott Vichinsky, Ali T. Taher, Aurelio Maggio, International Working Group on Thalassemia (IWG-THAL)

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Learning from conect4children: A Collaborative Approach towards Standardisation of Disease-Specific Paediatric Research Data by Anando Sen, Victoria Hedley, Eva Degraeuwe, Steven Hirschfeld, Ronald Cornet, Ramona Walls, John Owen, Peter N. Robinson, Edward G. Neilan, Thomas Liener, Giovanni Nisato, Neena Modi, Simon Woodworth, Avril Palmeri, Ricarda Gaentzsch, Melissa Walsh, Teresa Berkery, Joanne Lee, Laura Persijn, Kasey Baker, Kristina An Haack, Sonia Segovia Simon, Julius O. B. Jacobsen, Giorgio Reggiardo, Melissa A. Kirwin, Jessie Trueman, Claudia Pansieri, Donato Bonifazi, Sinéad Nally, Fedele Bonifazi, Rebecca Leary, Volker Straub

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