Jeanson, F.; Gibson, S. J.; Alper, P.; Bernier, A.; Woolley, J.P.; Mietchen, D.; Strug, A.; Becker, R.; Kamerling, P.; Sanchez Gonzalez, M.; Mah, N.; Novakowski, A.; Wilkinson, M.D.; Benhamed, O.M.; Landi, A.; Krog, G. P.; Müller, H.; Riaz, U.; Veal, C.; Holub, P.; van Enckevort, E.; Brookes, A. J., Getting your DUCs in a row - standardising the representation of Digital Use Conditions, Scientific Data, 11, 464 (2024), DOI 10.1038/s41597-024-03280-6

Tagarro, A.; Domínguez-Rodríguez, S.; Cotton, M.; Otwombe, K.;Klein, N.;Lain, M.G.; Nhampossa, T.; Maiga, A. I.; Barnabas, S.; Vaz, P.; Violari, A.; Fernández-Luis, S.; Behuhuma, O.; Sylla, M.; López-Varela, E.; Naniche, D.; Janse-Van-Rensburg, A.; Liberty, A.; Ramsagar, N.;Smit, T.; Makhari, S.; Ismael, N.; Giaquinto, C.;Rossi, P.; Kuhn, L.; Palma, P.; Spyer, M.; Lichterfeld, M.; Nastuoli, E.; Giannuzzi, V.; Ballestero, A.; Cotugno, N.; Morrocchi, E.; Oletto, A.; Traoré, F. T.;Dobbels, E.; Akhalwaya, Y.; Ording-Jespersen, G.; Foster, C.; Rabie, H.; Amuge, P.; Brehin, C.; Pahwa, S.; Coulibaly, Y. A.; Rojo, P., High mortality following early initiation of antiretroviral therapy in infants living with HIV from three African countries, Science direct.com

Felisi, M.; Bonifazi, F.; Toma, M.; Pansieri, C.;Leary, R.; Hedley, V.; Cornet, R.; Reggiardo, G.; Landi, A.; D’Ercole, A.; Salma Malik, S.; Nally, S.; Sen, A.; Palmeri, A.; Bonifazi, D.; Ceci, A., Mapping of Data-Sharing Repositories for Paediatric Clinical Research—A Rapid Review, MDPI 2024, 9 issue 4, DOI 10.3390/data9040059

Landi, A.; Mimouni, Y.; Giannuzzi, V.; Schaefer, F.; Altavilla, A., Gibson, S.; Daria Julkowska, D.; The creation of an adaptable informed consent form for research purposes to overcome national and institutional bottlenecks in ethics review: experience from rare disease registries, Front. Med. 2024, 11. DOI 10.3389/fmed.2024.1384026

Sen, A.; Hedley, V.; Degraeuwe, E.; Hirschfeld, S.; Cornet, R.; Walls, R.; Owen, J.; Robinson, P.N.; Neilan, E.G.; Liener, T.; Nisato, G.; Modi, N.; Woodworth, S.; Palmeri, A.; Gaentzsch, R.; Walsh, M.; Berkery, T.; Lee, J.; Persijn, L.; Baker, K.; An Haack, K.; Segovia Simon, S.; Jacobsen, J. O. B.; Reggiardo, G.; Kirwin, M. A.; Trueman, J.; Pansieri, C.; Bonifazi, D.; Nally, S.; Bonifazi, F.; Leary, R.; Straub, V.; Learning from conect4children: A Collaborative Approach towards Standardisation of Disease-Specific Paediatric Research Data, MDPI 2024, 9. DOI: 10.3390/data9040055

Giannuzzi, V.; Bertolani, A.; Torretta, S.; Reggiardo, G.; Toich, E.; Bonifazi, D.; Ceci, A. Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from paediatric perspective Front. Med. 2024, 11. DOI: 10.3389/fmed.2024.1369547

Ceci, A.; Kountouris, P.; Didio, A.; Bonifazi, F. Special Issue “Personalized Medicine in Blood Disease of Children” J. Pers. Med. 2024, 14, 285. DOI: 10.3390/jpm14030285

Mastrolorito, F.; Togo, M.V.; Gambacorta, N.; Trisciuzzi, D.; Giannuzzi, V.; Bonifazi, F.; Liantonio, A.; Imbrici, P.; De Luca, A.; Altomare, C.D.; Ciriaco, F.; Amoroso, N.; Nicolotti, O. TISBE: A Public Web Platform for the Consensus-Based Explainable Prediction of Developmental Toxicity Chem. Res. Toxicol. 2024, XX. DOI: 10.1021/acs.chemrestox.3c00310

Healy, P.; Verrest, L.; Felisi, M.; Ceci, A.; Della Pasqua, O. Dose rationale for gabapentin and tramadol in pediatric patients with chronic pain. Pharmacol Res Perspect. 2023;11:e01138. doi:10.1002/prp2.1138

Ceci A, Conte R, Didio A, Landi A, Ruggieri L, Giannuzzi V and Bonifazi F. Target therapy for high-risk neuroblastoma treatment:integration of regulatory and scientific tools is needed. Front. Med. 2023;10:1113460. doi: 10.3389/fmed.2023.1113460

Ruggieri, L.; Ceci, A.; Georgieva, B.; Bertolani, A.; Bartoloni, F.; Bonifazi, F.; Migliaccio, G.; Altavilla, A.; Della Pasqua, O.; Mikros, E.; Kubova, H.; Martelli, P.; Allegaert, K.; Dimitri, P.; Staud, F.; Smits, E.; Kleanthous, M.; Giannattasio, S.; Denora, N.; Tuleu, C.; Bonifazi, D. Collaborative approach to enhance the knowledge on paediatric rare diseases: the European Paediatric Translational Research Infrastructure. Meeting abstracts from the 11th edition of the European conference on Rare Diseases & Orphan Products (ECRD) 2022. Orphanet J Rare Dis 18 (Suppl 1), 118 (2023) P24. https://doi.org/10.1186/s13023-023-02707-4

de Wildt, S.N.; Foeldvari, I.; Siapkara, A.; Lepola, P.; Kriström, B.; Ruggieri, L.; Eichler, I.; Egger, G.F. Off-label is not always off-evidence: authorising paediatric indications for old medicines. Lancet Child Adolesc Health. 2023 Apr 25:S2352-4642(23)00083-4. doi: 10.1016/S2352-4642(23)00083-4

Kerpel-Fronius S, Giannuzzi V, Stoyanova-Beninska V, Sun Z. Reviews in: Regulatory Science. Front. Med. Sec. Regulatory Science. Volume 10 - 2023. doi: 10.3389/fmed.2023.1206753

Musuamba Tshinanu F, Cheung SYA, Colin P, Davies EH, Barret JS, Pappalardo F, Chappell M, Dogne JM, Ceci A, Della Pasqua O, Rusten IS. Moving towards a Question-centric approach for regulatory decision-making in the context of drug assessment. Clin Pharmacol Ther. 2023 Jan 27. doi: 10.1002/cpt.2856

Giannuzzi V, Ruggieri L, Conte R, Manfredi C, Felisi M, Kubiak C, Matei M, Malik S, Demotes J, Ceci A, Bonifazi D. PedCRIN tool for the biosamples management in paediatric clinical trials. Clin Transl Sci. 2023 Feb 9. doi: 10.1111/cts.13489

Giannuzzi V, Stoyanova-Beninska V and Hivert V (2022) Editorial: The use of real world data for regulatory purposes in the rare diseases setting. Front. Pharmacol. 13:1089033. doi: 10.3389/fphar.2022.1089033

Vitrano A, Musallam KM, Meloni A, Addario Pollina S, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A, International Working Group on Thalassemia. Random Forest Clustering Identifies Three Subgroups of β-Thalassemia with Distinct Clinical Severity. Thalassemia Reports. 2022; 12(1):14-23. https://doi.org/10.3390/thalassrep12010004

Sultana J, Crisafulli S, Almas M, Antonazzo IC, Baan E, Bartolini C, Bertuccio MP, Bonifazi F, Capuano A, Didio A, Ehrenstein V, Felisi M, Ferrajolo C, Fontana A, Francisca R, Fourrier-Reglat A, Fortuny J, Gini R, Hyeraci G, Hoeve C, Kontogiorgis C, Isgrò V, Lalagkas PN, L'Abbate L, Layton D, Landi A, Narduzzi S, Pereira LR, Poulentzas G, Rafaniello C, Roberto G, Scondotto G, Sportiello L, Toma M, Toussi M, Verhamme K, Volpe E, Trifirò G. Overview of the EU PAS register post-authorization studies performed in Europe from September 2010 to December 2018. Pharmacoepidemiol Drug Saf. 2022 Jan 29. doi: 10.1002/pds.5413

Moretti F, Ruiz F, Bonifazi F, Pizzo E, Kindblom JM; the c4c HTA expert group. Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project. Br J Clin Pharmacol. 2021 Dec 20. doi: 10.1111/bcp.15190. Epub ahead of print. PMID: 34931373

Dimitri P, Pignataro V, Lupo, M, Bonifazi D, Henke M, Musazzi UM, Ernst F, Minghetti P, Redaelli DF, Antimisiaris SG, Migliaccio G, Bonifazi F, Marciani L, Courtenay AJ, Denora N, Lopedota A. Medical Device Development for Children and Young People—Reviewing the Challenges and Opportunities. Pharmaceutics 2021, 13, 2178. https://doi.org/10.3390/pharmaceutics13122178

Turner M, Cheng K, de Wildt S, Hildebrand H, Attar S, Rossi P, Bonifazi D, Ceci A, Claverol J, Nafria B, Giaquinto C. European research networks to facilitate drug research in children. Br J Clin Pharmacol. 2020;1–9. DOI: 10.1111/bcp.14545.

Maggio A, Kattamis A, Felisi M, Reggiardo G, El-Beshlawy A, Bejaoui M, Sherief L, Christou S, Cosmi C, Della Pasqua O, Del Vecchio G, Filosa A, Cuccia L, Hassab H, Kreka M, Origa R, Putti M, Spino M, Telfer P, Tempesta B, Vitrano A, Tsang YC, Zaka A, Tricta F, Bonifazi D, Ceci A. Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial. The Lancet. Haematology. Jun 2020; 7(6):e469-e478. DOI: 10.1016/s2352-3026(20)30100-9

Altavilla A, Giannuzzi A, Lupo M, Bonifazi D, Ceci A. Ethical, legal and regulatory issues of paediatric translational research call for an adequate model of governance. European Journal of Health Law. May 2020. 1–19. https://doi.org/10.1163/15718093-BJA10010

Bonifazi F, Volpe E, Digregorio G, Giannuzzi V, Ceci A. Machine Learning Systems Applied to Health Data and System. European Journal of Health Law 27. 2020; 1-17. doi: 10.1163/15718093-BJA10009

Gini R, Sturkenboom M, Sultana J, Cave A, Landi A, Pacurariu A, Roberto G, Schink T, Candore G, Slattery J, Trifirò G, The Working Group 3 of ENCePP. Different strategies to execute multi-database studies for medicines surveillance in real world setting: a reflection on the European model. Clinical Pharmacology & Therapeutics. April 2020. doi:10.1002/cpt.1833

 Altavilla A, Herveg J, Giannuzzi V, Landi A, Ceci A. The Secondary Use of Paediatric Data Under GDPR: Looking for New Safeguards for Research. European Pharmaceutical Law Review. 2019; Volume 3, Issue 4.156 - 164. https://doi.org/10.21552/eplr/2019/4/6

Ruggieri L, Bonifazi D, Landi A, Bonifazi F, Bartoloni F, Costello M, Felisi MG, Gasthuys E, Godo A, Torres FM, Nadal D, Nuytinck L, Rocchi F, Turner M, Ceci A.. Survey by TEDDY European Network of Excellence for Paediatric Clinical Research demonstrates potential for Europe‐wide trials. Acta Paediatrica. 2019;00:1–6. doi: 10.1111/apa.15020

 Landi A, Thompson M, Giannuzzi V, Bonifazi F, Labastida I,  Bonino da Silva Santos LO, Roos M. The “A” of FAIR – As open as possible, as closed as necessary. Data Intelligence. 2020; 47–55. doi: 10.1162/dint_a_00027

Nori T, Ghazisaeedi M, Aliabad GM, Mehdipour Y, Mehraeen E, Conte R, Safdari R, International Comparison of Thalassemia Registries: Challenges and Opportunities. ACTA INFORM MED. 2019 MAR 27(1): 58-63, doi: 10.5455/aim.2019.27.58-63

Ceci A, Conte R, Didio A, Bonifazi D, Felisi M, Giannuzzi V, Bonifazi F. An overview of the efficacy and safety of deferiprone in paediatric patients with congenital haemoglobinopathies and chronic iron overload. Expert Opinion on Orphan Drugs. 2019; 7:4,181-197. doi: 10.1080/21678707.2019.1613977

Giannuzzi V. Abandoned OMPs. 9th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018). Orphanet Journal of Rare Diseases. 2018;13(S2)

Altavilla A. Safe, Innovative and Accessible Medicines in Europe: A Renewed Strategy for Patients and the Pharmaceutical Sector. European Journal of Health Law. 2018;25(2):133-156. DOI: 10.1163/15718093-12520381

Giannuzzi V, Landi A, Bartoloni F, Ceci A. A Review on Impact of General Data Protection Regulation on Clinical Studies and Informed Consent. J Clin Res Bioeth 2018; 9(3):327. doi: 10.4172/2155-9627.1000327

Veal GJ, Malik S, Lupo M, MacFarlane S, Lepola P, Costello M, Ceci A, Boué C, Lecour C, Otto A, Rastegari M, Berry P. Investigating the roles and training of paediatric research nurses working across Europe: a questionnaire-based survey. BMJ Paediatr Open. 2017 Sep 5;1(1):e000170 doi:  10.1136/bmjpo-2017-000170

Giannuzzi V, Landi A, Bosone E, Giannuzzi F, Nicotri s, Torrent-Farnell J, Bonifazi F, Felisi M, Bonifazi D, Ceci A. Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs. BMJ Open. 2017 Sep 11;7(9):e017358. doi: 10.1136/bmjopen-2017-017358

Neubert A, Baarslag MA, van Dijk M, van Rosmalen J, Standing JF, Sheng Y, Rascher W, Roberts D, Winslade J, Rawcliffe L, Hanning SM, Metsvaht T, Giannuzzi V, Larsson P, Pokorná P, Simonetti A, Tibboel D on behalf of CLOSED Consortium. The CLOSED trial; CLOnidine compared with midazolam for SEDation of paediatric patients in the intensive care unit: study protocol for a multicentre randomised controlled trial. BMJ Open. 2017 Nov 8;7(11):e016031corr1. PMID: 28637741; PMCID: PMC5726127.

Giannuzzi V, Conte R, Landi A, Ottomano SA, Bonifazi D, Baiardi P, Bonifazi F, Ceci A. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet J Rare Dis.2017 Apr 3;12(1):64

Giannuzzi V, Devlieger H, Margari L, Odlind V, Ragab L, Bellettato C et al. The ethical framework for performing research with rare inherited neurometabolic disease patients. Eur J Pedatr. 2017 Mar;176(3):395-405

Bonifazi F, Conte R, Baiardi P, Bonifazi D, Felisi M, Giordano P, Giannuzzi V, Iacono A, Padula R, Pepe A, Putti MC, Ruggieri L, Del Vecchio GC, Filosa A, Maggio A, Ceci A on behalf of the HTA-THAL Multiregional Registry.  Pattern of complications and burden of disease in patients affected by beta thalassemia major. Curr Med Res Opin. 2017 33(8):1525-1533

D'Andria P, Giannuzzi V, Bonifazi F, Ceci A, Denora N, Altomare C. Formulating Medicines for children: challenges and issues. La Chimica e l'Industria (3): 2016

Conte R, Ruggieri L, Gambino A, Bartoloni F, Baiardi P, Bonifazi D, Bonifazi F, Felisi M, Giannuzzi V, Padula R, Pepe A, Putti MC, Del Vecchio GC, Maggio A, Filosa A, Iacono A, Mangiarini L, Ceci A. The Italian multiregional thalassemia registry: Centers characteristics, services, and patients' population. Hematology. 2016 Aug;21(7):415-24

Giannuzzi V, Altavilla A, Ruggieri L, Ceci A. Clinical Trial Application in Europe: What Will Change with the New Regulation? Sci Eng Ethics. 2016 Apr;22(2):451-66

Botzenhardt S, Sing CW, Wong IC, Chan GC, Wong LY, Felisi M, Rascher W, Ceci A, Neubert A. Safety Profile of Oral Iron Chelator Deferiprone in Chinese Children with Transfusion-Dependent Thalassaemia. Curr Drug Saf. 2016;11(2):137-44

Cassis L, Cortès-Saladelafont E, Molero-Luis M, Yubero D, González MJ, Ormazábal A, Fons C, Jou C, Sierra C, Ponce EC, Ramos F, Armstrong J, O'Callaghan MM, Casado M, Montero R, Meavilla-Olivas S, Artuch R, Barić I, Bartoloni F, Bellettato CM, Bonifazi F, Ceci A, Cvitanović-Šojat L, Dali CI, D'Avanzo F, Fumic K, Giannuzzi V, Lampe C, Scarpa M, Garcia-Cazorla Á Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders. Orphanet J Rare Dis. 2016; 11: 147. doi:  10.1186/s13023-016-0431-1dis

Ruggieri L, Giannuzzi V, Baiardi P, Bonifazi F, Davies EH, Giaquinto C, Bonifazi D, Felisi M, Chiron C, Pressler R, Rabe H, Whitaker MJ, Neubert A, Jacqz-Aigrain E, Eichler I, Turner MA, Ceci A; on behalf of the GRiP Consortium. Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines. Eur J Pediatr. 2015; 174(4):481-91

Lorusso S, Chiarolla E, Bonifazi F, Casino D, Sassano S. La Rete regionale degli acquisti della Regione Basilicata dall’Health technology assessment alla spesa: presentazione di un case study. Politiche Sanitarie. 2015;16(2):139-149

Ceci A, Mangiarini L, Bonifazi F, Conte R. Multidisciplinary care in haemoglobinopathies. Thalassemia Reports. 2014; 4:4875

Maggio A, Filosa A, Vitrano A, Aloj G, Kattamis A, Ceci A, Fucharoen S, Cianciulli P, Grady RW, Prossomariti L, Porter JB, Iacono A, Cappellini MD, Bonifazi F, Cassarà F, Harmatz P, Wood J, Gluud C. Iron chelation therapy in thalassemia major: A systematic review with meta-analyses of 1520 patients included on randomized clinical trials. Blood Cells Mol Dis. 2011 Oct 15;47(3):166-75

Ceci A, Mangiarini L, Felisi M, Bartoloni F, Ciancio A, Capra M, D'Ascola D, Cianciulli P, Filosa A. The management of iron chelation therapy: preliminary data from a national registry of thalassaemic patients. Anemia. 2011;2011:435683

Scarpa M, Ceci A, Tomanin R, Mincarone P, Begley D. Personalised medicine in paediatrics: individualising treatment in children with rare neurological diseases. EPMA J. 2011 Jun; 2(2): 231–239

Bonerba E, Ceci E, Conte R, Tantillo G. Survey of the presence of patulin in fruit juices. Food Addit Contam Part B Surveill. 2010;3(2):114‐9

Bonerba E, Conte R, Ceci E, Tantillo G. Assessment of dietary intake of patulin from baby foods. J Food Sci. 2010 Sep;75(7):T123‐5

Trama A, Pierannunzio D, Loizzo A, Taruscio D, Ceci A. Availability of medicines for rare diseases in EU countries. Pharmaceuticals Policy and Law, 2009;11(1,2):101-9

Baiardi P, Girotto S, Della Pasqua O, Harper L, Grosch-Worner I, Ceci A, Giaquinto C. Clinical trials for paediatric medicines in Europe. Pharmaceuticals Policy and Law, 2009;11(1,2):71-78

Catapano M, Manfredi C, Paolucci P, Cross H, Verhamme K, Mellado Pena MJ, Grosch-Worner I, Knibbe C, Ceci A. Recommendation for drug development for children. Pharmaceuticals Policy and Law, 2009;11(1,2):61-70

Krekels E, Ceci A, Iolascon A, Girotto S, Della Pasqua O. The role of paediatric pharmacogenetic studies in Europe. Pharmaceuticals Policy and Law, 2009;11(1,2):23-30

Neubert A, Felisi M, Bonifazi A, Manfredi C, Wong ICK, Ceci A. Off-label and unlicensed use of medicines for children. Pharmaceuticals Policy and Law, 2009;11(1,2):41-49

Neubert A, Wong IC, Bonifazi A, Catapano M, Felisi M, Baiardi P, Giaquinto C, Knibbe CA, Sturkenboom MC, Ghaleb MA, Ceci A. Defining off-label and unlicensed use of medicines for children: Results of a Delphi survey. Pharmacol Res. 2008 Nov-Dec;58(5-6):316-22

Benzi G, Ceci A. Drugs and food supplements in sports. The nandrolone lessons. Minerva Pediatr. 2001 Oct;53(5):395-6

Benzi G, Ceci A. Creatine as nutritional supplementation and medicinal product. J Sports Med Phys Fitness. 2001 Mar;41(1):1-10