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Articles in Journal
2024

Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective

Giannuzzi V, Bertolani A, Torretta S, Reggiardo G, Toich E, Bonifazi D and Ceci A

Front. Med. 11:1369547. doi: 10.3389/fmed.2024.1369547

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2022

European research networks to facilitate drug research in children

Mark A TurnerKatharine ChengSaskia de WildtHeidrun HildebrandSabah AttarPaolo RossiDonato BonifaziAdriana CeciJoana ClaverolBegonya NafriaCarlo Giaquinto

Br J Clin Pharmacol 2022 Oct;88(10):4258-4266. doi: 10.1111/bcp.14545. Epub 2020 Sep 20.

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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

Lederer C.W, Koniali L, Buerki-Thurnherr T, Papasavva P.L, La Grutta S, Licari A, Staud F, Bonifazi D, Kleanthous M.

Pharmaceutics 2022, 14, 793. doi.org/10.3390/pharmaceutics14040793

 

2021

Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities

P. Dimitri, V. Pignataro, M. Lupo, D. Bonifazi , M. Henke , U. M. Musazzi, F. Ernst, P. Minghetti, D. F. Redaelli, S. G. Antimisiaris, G. Migliaccio, F. Bonifazi, L. Marciani, A. J. Courtenay, N. Denora, A. Lopedota

Pharmaceutics 2021, 13(12), 2178;  https://doi.org/10.3390/pharmaceutics13122178

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The GluN2B-Selective Antagonist Ro 25-6981 Is Effective

P. Mareš, L. Kozlová , A. Mikulecká, H. Kubová

Pharmaceutics 2021, 13, 1482; https://doi.org/10.3390/pharmaceutics13091482

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Children’s Preferences for Oral Dosage Forms and Their Involvement in Formulation Research via EPTRI (European Paediatric Translational Research Infrastructure)

E. Alessandrini, F. Brako, M. Scarpa, M. Lupo , D. Bonifazi, V. Pignataro, M. Cavallo, O. Cullufe, C. Enache, B. Nafria, J. Claverol, L. De Taeye, E. Vermeulen, J. Preston, C. Tuleu

Pharmaceutics 2021, 13, 730; https://doi.org/10.3390/pharmaceutics13050730

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Detection of Breathing Movements of Preterm Neonates by Recording Their Abdominal Movements with a Time-of-Flight Camera

F. C. Wiegandt, D. Biegger , J. F. Fast, G. Matusiak, J. Mazela, T. Ortmaier, T. Doll, A. Dietzel, B. Bohnhorst, G. Pohlmann

Pharmaceutics 2021, 13, 721; https://doi.org/10.3390/pharmaceutics13050721

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Drug Safety in Translational Paediatric Research: Practical Points to Consider for Paediatric Safety Profiling and Protocol Development: A Scoping Review

B. Aurich, E. Jacqz-Aigrain

Pharmaceutics 2021, 13, 695; https://doi.org/10.3390/pharmaceutics13050695

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Breath-Triggered Drug Release System for Preterm Neonates

F. C. Wiegandt, U. P. Froriep, F. Müller, T. Doll, A. Dietzel, G. Pohlmann

Pharmaceutics 2021, 13, 657; https://doi.org/10.3390/pharmaceutics13050657

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Human Microbiome in Children, at the Crossroad of Social Determinants of Health and Personalized Medicine

Talía Sainz, Valeria Pignataro, Donato Bonifazi, Simona Ravera, María José Mellado, Antonio Pérez-Martínez , Adela Escudero, Adriana Ceci and Cristina Calvo

Children 2021, 8, 1191; https://doi.org/10.3390/children8121191

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The Neonatal and Juvenile Pig in Pediatric Drug Discovery and Development

M. Ayuso, L. Buyssens, M. Stroe, A. Valenzuela, K. Allegaert, A. Smits, P. Annaert, A. Mulder, S. Carpentier, C. Van Ginneken, S. Van Cruchten

Pharmaceutics 2021, 13/3; doi: 10.3390/pharmaceutics13030415

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Patient-Derived Induced Pluripotent Stem Cells (iPSCs) and Cerebral Organoids for Drug Screening and Development in Autism Spectrum Disorder: Opportunities and Challenges

C. Villa, R. Combi, D. Conconi, M. Lavitrano

Pharmaceutics 2021, 13/2; doi: 10.3390/pharmaceutics13020280

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2020

Involve Children and Parents in Clinical Studies

Eric VermeulenKim KarsenbergJohanna H van der LeeSaskia N de Wildt

 
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Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance

A. Altavilla, V. Giannuzzi, M. Lupo, D. Bonifazi, A. Ceci

European Journal of Health Law 2020, May 18; doi: 10.1163/15718093-bja10010

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Pediatric Biobanking: Kids Are Not Just Little Adults

D. R. Catchpoole, D. Carpentieri, S. Vercauteren, L. Wadhwa, W. Schleif, L. Zhou, J. Zhou, R. M. Labib, E. Smits, E. H. Conradie

Biopreservation and Biobanking 18/4, 2020, August; doi: 10.1089/bio.2020.29071.djc

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2018

Drug evaluation in children 10years after the European pediatric regulation current challenges and perspectives

Valery Élie, Stéphanie LerouxFlorentia KaguelidouEvelyne Jacqz-Aigrain

Therapie. 2018 Apr;73(2):113-117. doi: 10.1016/j.therap.2017.11.006. Epub 2018 Feb 17.

 

 

 

 

Chapter in book

Challenges and New Frontiers in the Paediatric Drug Discovery and Development

A. Intini, D. Bonifazi, G. Migliaccio

Drug Discovery and Development – New Advances, 2019; 10.5772/intechopen.85635

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Preface: overview of pediatric drug development and pharmacovigilance

Elke Gasthuys, Lien Dossche, Karel Allegaert, Mark A Turner

Essentials of Translational Pediatric Drug Development – From Past Needs to Future Opportunities, 2024; Paperback ISBN: eBook ISBN: 

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The needs, challenges and opportunities from the academic researchers’ perspective

Elke Gasthuys, Karel Allegaert, Eva Degraeuwe, Hidefumi Nakamura, Johan Vande Walle, Gregory Kearns

Essentials of Translational Pediatric Drug Development – From Past Needs to Future Opportunities, 2024; Paperback ISBN: eBook ISBN: 

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Application of preclinical juvenile animal models

Eline Hermans, Jaan Toelen, Domenico Ventrella, Luc De Schaepdrijver, Katie Turner, Siska Croubels, Mathias Devreese

Essentials of Translational Pediatric Drug Development – From Past Needs to Future Opportunities, 2024; Paperback ISBN: eBook ISBN: 

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Performing clinical drug trials in acute and critically ill neonates and children

Pieter A De Cock, Anne Smits, Mehdi Oualha, Gideon Stitt, Tuuli Metsvaht, Nori Smeets, Karel Allegaert, Mark A Turner, Angela Amigoni, Athena F Zuppa, Saskia N de Wildt

Essentials of Translational Pediatric Drug Development – From Past Needs to Future Opportunities, 2024; Paperback ISBN: eBook ISBN: 

EPTRI Special Issues

Special Issue entitled “Supporting the Pediatric Drug Development: From Basic Research to Clinical Studies and Technological Advancements” has been published in the Frontiers in Paediatrics Journal at the end of 2023 and beginning of 2024.

The Special Issue aiming to contribute to the international progress in paediatric research by supporting and facilitating the publication of high-quality studies on a variety of aspects of drug development that can have an impact on children’s therapeutics and health, has been addressed to all researchers and partners involved in the development of paediatric medicines working on the following topics:

1. Basic and pre-clinical research that provides new knowledge on human growth and ontogeny influencing drugs effects and drug development
2. Research on new biomarkers targeting common or rare paediatric diseases or diseases that can affect children as well as adults
3. Research on technologies, standardised models, and analytical tools/IT tools that can be applied in a paediatric setting (drugs and medical devices)
4. Modelling, simulation, and extrapolations methods applied to paediatric medicines developmental phases mainly devoted to the right dose identification and risk/benefit preclinical evaluation.
5. Innovative paediatric-tailored formulations, including 3D applications
6. Investigation & scrutiny of clinical and RWD data on the use of medicines in children and adolescents.
7. Research on the ethical and legal framework for paediatric studies including the consenting process, raising awareness and improving patients engagement in paediatric research

Eight articles were published in this Special Issue.

  • Paediatric medicine issues and gaps from healthcare workers point of view: survey results and a narrative review from the global accelerator for paediatric formulations project, Elisa Barbieri, Chiara Minotti, Sara Cavagnis, Carlo Giaquinto, Bernadette Cappello, Martina Penazzato and Marc Lallemant. Front. Pharmacol. 2023 14:1200848. doi: 10.3389/fphar.2023.1200848
  • Impact of vaccination with different types of rotavirus vaccines on the incidence of intussusception: a randomized controlled meta-analysis, Wang G, Zhang K, Zhang R, Kong X and Guo C. Front. Pharmacol. 2023 11:1239423. doi: 10.3389/fped.2023.1239423
  • In vitro intestinal simulation system on the carbon source utilization characteristics and mechanism of interspecific syntrophic effects of Bifidobacterium longum CECT7894 and Pediococcus pentosaceus CECT8330, Li J, Xu L, Liao J, Li X, Wang X, Wu Q and Zhu L. Front. Pharmacol. 2023 11:1276846. doi: 10.3389/fped.2023.1276846
  • Pragmatic physiologically-based pharmacokinetic modeling to support clinical implementation of optimized gentamicin dosing in term neonates and infants: proof-of-concept, de Hoop-Sommen MA, van der Heijden JEM, Freriksen JJM, Greupink R and de Wildt SN. Front. Pharmacol. 2023 11:1288376. doi: 10.3389/fped.2023.1288376
  • Development and performance of the c4c national clinical trial networks for optimizing pediatric trial facilitation, Degraeuwe E, van der Geest T, Persijn L, Nuytinck L, Raes A, Turner M, Fernandes RM, Vande Walle J, de Wildt SN and IMI2 project conect4children(c4c) consortium, including National Hubs/Networks Belgian Pediatric Clinical Research Network (BPCRN) (Belgium) and Pedmed-L (Netherlands). Front. Pharmacol. 2023 11:1302272. doi: 10.3389/fped.2023.1302272
  • Comparison of remimazolam and propofol combined with low dose esketamine for pediatric same-day painless bidirectional endoscopy: a randomized, controlled clinical trial, Chu T, Zhou S, Wan Y, Liu Q, Xin Y, Tian Z, Yan T and Xu A. Front. Pharmacol. 2024 15:1298409. doi: 10.3389/fphar.2024.1298409
  • Evaluating palatability in young children: a mini-review of relevant physiology and assessment techniques, Schluterman HM, Linardos CG, Drulia T, Marshall JD and Kearns GL. Front. Pharmacol. 2024 12:1350662. doi: 10.3389/fped.2024.1350662
  • Piperacillin/tazobactam treatment in children: evidence of subtherapeutic concentrations, Ye P, Shi J, Guo Z, Yang X, Li Q, Chen K, Zhao F, Zhou H, Zhang Y, van den Anker J, Song L and Zhao W. Front. Pharmacol. 2024 15:1254005. doi: 10.3389/fphar.2024.1254005

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Special Issue dedicated to the Scientific Highlights in the European Paediatric Translational Research Infrastructure has been published into the section “Drug Delivery and Controlled Release” of the Pharmaceutics Journal at the end of 2021.  

The Special Issue has been addressed to all researchers and partners involved in the development of paediatric medicines working on the following topics: i) paediatric medicines drug targets and methods of identification; ii) advanced cellular/in vitro models for paediatric drug targeting; iii) paediatric formulations and toxicology of drugs; iv) drug delivery design in paediatrics; v) translational research. 

The EPTRI members published 12 articles in this Special Issue.