18 July – Presenters & Presentations
Karel Allegaert
Karel Allegaert, MD, PhD is pediatrician-neonatologist and clinical pharmacologist. He is Professor at KU Leuven and Consultant at Erasmus MC Rotterdam. His clinical research has focused mainly on perinatal and developmental pharmacology, with a specific interest in perinatal and neonatal clinical pharmacology and the relevant co-variates (maturational, non-maturational, pharmacogenetics, disease-related, pregnancy-related) of pharmacokinetics, and the subsequent use of in vivo datasets to develop prediction models. More recently, his research line has further expanded to in vitro models (placenta, renal tubular cells), in vivo models (bronchopulmonary dysplasia rabbit model) and ng term pharmacovigilance in former preterm neonates. He has also expertise with DSMB activities, and consultancies to individual companies or authorities, and is involved in several international networks like IMIc4c, the International Neonatal Consortium, and learned societies (ESPR, IUPHAR) or editorial boards of relevant journals to the topic.
Marek Migdal
Marek Migdał, MD, PhD, has been graduated from the Medical University of Warsaw in 1979. Since October 1, 1979, he is employed at the Children’s Memorial Health Institute. Initially, he worked in the Department of Paediatrics, then from 1986 in the Department of Anaesthesiology and Intensive Care. In December 2018, he was appointed by the Minister of Health to the position of the Director of the CMHI. In 1985-1986 and in 1990 he did research internships in France (SEPCR scholarship, then French government scholarship). In 2005-2009, he was the Head of the PICUt, then, until 2018, the Deputy head of the PICU. He is a paediatrician, specialist in lung diseases. In 1990, he defended his doctoral dissertation at the CMHI in the field of lung function measurement in newborns and infants with respiratory failure. He is a researcher (assistant professor) at the Children’s Memorial Health Institute, author of more than 230 publications, lecturer at postgraduate courses, organizer of a number of conferences dedicated to paediatric intensive care and clinical trials in Poland. Active coordinator of international scientific cooperation. He was the Polish coordinator of the following European grants: EU-CHS – European network for central hypoventilation syndrome (2008-2012), GRIP- Global Research in Paediatrics (2011-2017), in 2016-2018 he was the European coordinator of the SMART grant (Small Medicines Advanced Research Training) and the coordinator of the Polish grants ID-EPTRI (European Translational Research Infrastructure) and c4c (conect4children – a pan-European clinical trial network).
Leanne West
Leanne West is Chief Engineer of Pediatric Technologies at the Georgia Institute of Technology and Pediatric Innovation Catalyst at the Global Center for Medical Innovation where she leads innovation in pediatric medical devices. In her 25+ years working at Georgia Tech, she has led multimillion dollar programs and teams of researchers to develop products for government and industry partners. She also started her own company, Intelligent Access, to take her invention of a wireless personal captioning system to market. She serves as the technical liaison between Georgia Tech and pediatric hospitals around the world, with Children’s Healthcare of Atlanta and Shriners Hospitals being the main partners. Ms. West works closely with clinicians to understand and identify problems that need a solution to allow them to take better care of their patients. She is an invited Judge for many medical device pitch competitions and serves on several Boards in the healthcare and technology arenas. West is the President of the International Children’s Advisory Network (iCAN). Since 2014, iCAN fosters greater global understanding about the importance of the pediatric patient and caregiver voice in healthcare, clinical trials, and research.
Ian Wong
Professor Ian Wong is the former Head of Department of Pharmacology and Pharmacy, University of Hong Kong. He is also the holder of Lo Shiu Kwan Kan Po Ling Endowed Professorship in Pharmacy. As an academic pharmacist with research expertise in big data analytics and neuropsychopharmacology. He has led many research programmes in the UK, European and Hong Kong in ADHD. Professor Wong has over 600 publications with publication in prominent journals including Nature portfolio journals, JAMA Network Journals, The Lancet family journals, BMJ journals. According to Clarivate Analytics’ Essential Science Indicators, Professor Wong has been a top 1% scholar in pharmacology and pharmacy research from 2015 to 2023. In recognition of his research, Professor Wong has received numerous awards for his work as an academic pharmacist. He was awarded the Chemist and Druggist Pharmacy Practice Research Conference Medal from the Royal Pharmaceutical Society of Great Britain in 2004, an Honorary Fellowship from the Royal College of Paediatrics and Child Health in the UK in 2011, an Honorary Fellowship from the College of Pharmacy Practice in Hong Kong in 2013, and a Fellowship from the Royal Pharmaceutical Society in 2013, a Fellowship from British Pharmacological Society and International Society of Pharmacovigilance in 2020.
Samuele Cortese
Samuele Cortese is currently NIHR Research Professor, Professor of Child and Adolescent Psychiatry at the University of Southampton, and Honorary Consultant for Solent NHS Trust. Prof. Cortese’s main research interests are around neurodevelopmental disorders. He has published more than 350 peer-review papers. He has secured funding as main/co-applicant for more than £ 13 M. In 2022, he was included in the top 1% scientists in the field of psychiatry/psychology (Web of Science). Since 2022, he ranked #1 worldwide in terms of expertise on ADHD (Expertscape).
Viviana Guiannuzzi
PharmD, she has a PhD in cellular biochemistry and pharmacology, and a post-graduate degree master in Clinical Research of Medicines. Her main areas of expertise are: ethics and regulatory, informed consent and assent, health data processing for research purposes, European medicine regulatory procedures, R&D for rare and paediatric diseases. She performed non-clinical research in rare diseases, and she was the Secretariat Scientific Assistant of the Ethics Committee in Lecce (IT). Currently, at Fondazione per la Ricerca Farmacologica Gianni Benzi onlus she coordinates the research department and participates in EU and national research projects, where, in most of them, she leads the ethics and regulatory activities. She is a member representing patient’ organisations of Paediatric Committee, European Medicine Agency (EMA). She is/was member of international and national working groups (Ethics Working Group of EnprEMA; Working Group on Rare Cancers of ECPC (European Cancer Patient Coalition); paediatric pharmacology Working Group of the Italian Society of Pharmacology). Author/co-author of peer reviewed publications and abstracts, reviewer for indexed journals and speaker at national and international congresses/meetings in the relevant fields of expertise.
Giovanni Migliaccio
Giovanni Migliaccio is the Scientific Director of the Consorzio per Valutazioni Biologiche e Farmacologiche, (CVBF Pavia, IT) and a Senior Advisor for the European Translational Medicine Research Infrastructure (EATRIS, Amsterdam NL). He obtained a PhD in Biological science in 1977 at the University of Naples (Italy). His interest in science developed around stem cell proliferation and differentiation mechanisms. He spent two years as Post Doc at the University of Washington (Seattle, WA) and 5 years as Associate Scientist at the New York Blood Center (New York, NY). He continued his studies at the Istituto Superiore di Sanità (ISS) in Rome resulting in more than 150 scientific publications over the years. He is involved in the regulatory aspects of using cultured cells as part of human therapies and acts as an expert for the Italian government in the area of Cell and Gene Therapies. He is also an expert for these products for the European Medicines Agency (EMA). He was also the Chair of the CTP group at the European Department for Quality of Medicines (EDQM) from 2010 to 2018.
Marios Phylactides
Dr Marios Phylactides obtained his BsC in biochemistry from Imperial College, London in 1993. He completed his PhD studies in immunology at University college London in 1998. During his first post-doctoral position at John Radcliff Hospital, Oxford, he worked in the field of cystic fibrosis. After completing an MBA at London Business School, he joined the Molecular Genetics Thalassaemia Department at the Cyprus Institute of Neurology and Genetics (CING) in 2005. Dr Phylactides’ research interests include the identification of diagnostic biomarkers for liver fibrosis in β-thalassaemia, investigation of the regulatory mechanisms of γ-globin gene regulation, genotype/phenotype correlation studies for α- and β-thalassaemias and more recently the development of gene therapy vectors for α-thalassaemia. He has been lecturing at the CING Post Graduate School for the last ten years and has overseen the successful completion of the studies of two PhD students and seven MSc students. Dr Phylactides has published 30 papers in peer-reviewed journals, most of them in the field of haemoglobinopathies, with over 1500 citations and an h index of 15.
Julia Macente
Julia Macente is a pharmacist graduated from the State University of Maringá, Brazil in 2018. She holds a Master’s degree in Pharmaceutical Sciences from the State University of Maringá, Brazil, obtained in 2021. During her Master’s, she developed Physiologically-Based Pharmacokinetic (PBPK) models to evaluate the safety of levetiracetam doses in the pediatric population. Currently, she is pursuing a PhD at KU Leuven, Belgium (2021-present). Her research is part of the IMI project ConcePTION and focuses on developing PBPK models to assess drug exposure in the perinatal population.
Donato Bonifazi
Dr Donato Bonifazi is an Economist specialised in European Project Management and Chief Executive Officer at CVBF, a non-profit research consortium with 20 years’ experience in health and pharmaceutical research. He is Chair of the Events & Training WG at EUCROF (European CRO Federation), Board Member at TEDDY – European Network of Excellence for Paediatric Research, Member of the Coordinating Group at Enpr-EMA, Member of the ACT EU multi-stakeholder platform Advisory Group, Member of the iCAN – International Children Advisory Network Advisory Group. He has been project coordinator and trial manager of the multinational paediatric clinical trials EU-funded project DEEP – DEferiprone Evaluation in Paediatrics (FP7 GA 241683), involving more than 20 clinical centers from European and Mediterranean countries, and is involved in several projects aimed at enhancing research for paediatric and rare diseases patients: c4c – Network for European Clinical Trials for Children (H2020 GA 777389); EJP RD – European Joint Programme on Rare Diseases (H2020 GA 825575). He is Coordinator of EPTRI (European Paediatric Translational Research Infrastructure) aimed to create the framework for a new Research Infrastructure (RI) intended to enhance technology-driven paediatric research in drug discovery and early development phases to be translated into clinical research and paediatric use of medicines, diagnostics and devices, grouping more than one hundred research centers form 25 EU and not EU Countries. In March 2024, the EPTRI AISBL has been established in Leuven, Belgium, and the OrphaDev4Kids project has been selected by the EU to foster the development of paediatric medical devices for the treatment of children with rare diseases.
Lucia Ruggieri
Lucia has a master’s degree in medical biotechnology and a post-graduate Master of Science in Paediatric Medicines Development and Evaluation and in Biostatistics for Clinical Research. She is currently at the Benzi Foundation, where she is involved in scientific, methodological, regulatory and ethical activities related to paediatric clinical research and biobank-based research and collaborates in the scientific coordination and management of several international projects dealing with paediatric research and haemoglobinopaties.
She is a member of the EPTRI Central Management Office.
Giorgio Reggiardo
Giorgio has a Master’s Degree in Biological Science from the University of Genoa and a PhD in Medical Statistics and Epidemiology from the University of Pavia (Italy). Currently he is the Head of Health Research Project and Biostatistics Unit at CVBF. He is trained in epidemiology, biostatistics, multivariate analysis, Bayesian inference, meta-analysis, “big data” analytics, and clinical trial design. Since 1997 he has served as lead statistician for Phase I-IV clinical trials to provide statistical support for study design and statistical analysis, including pharmaceutical industry-sponsored successful Marketing Authorizations (AIC) submissions. He has expertise in the design and analysis of clinical trials, controlled randomized trials, power calculations, generalized estimating equations, survival analysis, and regression methodology. He wrote SAS Macros and R functions that carry out data management and statistical procedures, and he presented statistical methodological work in several professional conferences. Since 2012 he has been involved as a statistician in two International Pharmacovigilance Department for the detection of emerging signals of interest for pharmacovigilance (Statistical approach to Signal Detection Analysis). Since 2013 he has been actively involved in the Italian CDISC User Network group in collaboration with IBiG (Italian Biostatistics Group) and SAS Institute.
Nathan Ali
Mr. Nathan Ali earned his Bachelor of Science in Engineering and a Master of Science in Engineering from the University of Illinois in Agricultural & Biological Engineering. Post-graduation, Mr. Ali embarked on a dynamic career path, assuming roles spanning engineering, prototype development, electronics, programming, manufacturing, international technical sales, and business development. Founding Skygenic, Mr. Ali assembled a skilled team comprising front and back-end developers, cloud specialists, bioinformaticians, and physician scientists, united by a shared vision. Together, they have successfully engineered a cutting-edge multi-omic management platform tailored to meet the needs of academic institutions and scientists worldwide, positioning themselves as a global force in scientific advancement. Recognized for his entrepreneurial prowess and innovative endeavors, Mr. Ali has gained acceptance into the prestigious MassChallenge Accelerator Program, providing vital support for healthcare technology startups. Additionally, he has been selected for the McDermott Will & Emery Rise Program, affording legal assistance and resources tailored to minority-owned startups pioneering transformative solutions with global impact. Further showcasing his commitment to continuous learning and professional development, Mr. Ali has secured a scholarship to partake in the Tuck Executive Education at Dartmouth/Mass Mutual Diverse Business Program, underscoring his dedication to fostering inclusive growth and driving positive change on a global scale.
Franco Bartoloni
Franco has over twenty-five years of experience in Health Technology Management, Project Management, IT Management of disease/population registries and eCRFs, IT platforms manager in many international projects. Software Developer, Expert in data protection and privacy, Certified Web Marketing Specialist. Consultant at Fondazione Gianni Benzi where he is involved in Project management, IT solution design, Data Protection and Privacy.
Fedele Bonifazi
Fedele (Duccio) Bonifazi is the President of the Fondazione per la ricerca farmacologica Gianni Benzi onlus and Head of its IT & Research Laboratory. His background includes a master degree in bio-engineering and a post-graduate master in HTA. He is Project Manager for the “Study supporting the Impact Assessment of the revision of the EU legislation on medicines for children and rare diseases”, Project Coordinator of ARISE – African Research and Innovative Initiative for Sickle Cell Education: Improving Research Capacity for Service Improvement (EU GA no. 824021), “Data coordinating centre and data quality standards” work package co-leader and member of the c4c Steering Committee (IMI2-JU GA no. 777389) and member of the HTA working group of the Regional Health Agency AReSS Puglia (IT).
Oscar Della Pascua
Prof Oscar Della Pasqua is Chair in Clinical Pharmacology & Therapeutics at University College London, UK. He is also Senior Director of Clinical Pharmacology Modelling and Simulation at GlaxoSmithKline R&D and Associate Fellow at the National Research Council in Rome, Italy addition to his extensive experience in early and late clinical development, he leads a research group focused on biomarkers, disease modelling, and clinical trial design methodology. He has > 150 scientific publications in peer-reviewed journals and is Senior Editor of the British Journal of Clinical Pharmacology.
Alessandra Mezzelani
Alessandra Mezzelani has a PhD in Veterinary Biotechnologies and currently she is a staff researcher at the National Research Council, Institute of Biomedical Technologies (CNR-ITB), Segrate, Milan, Italy. Previously she was an associated researcher at National Institute of Tumors, Milan, Italy (1995-2001), a PhD student at ETH Zurich, Switzerland and CNR-IBBA, Milan, Italy (1992-1995) and a fellow at CNR-IBBA, Milan, Italy (1989-1992). Research Fields and activities: “Multi-omics” research in autism. She leads the CNR Unit of the European GEMMA Project https://www.gemma-project.eu/ . She is responsible of the Single Cell Analysis (SCA) Unit and is applying SCA to breast cancer, NAFLD and viral infections. She represents CNR-ITB at the EU LifeTime Initiative to improve European healthcare through cell-based interceptive medicine. She is responsible of the Translational Bioinformatics Lab. to produce “omics” data or to validate bioinformatic results and predictions. She has great experience in the usage of the common molecular biology techniques (RT-PCR, digital PCR, microarrays, Illumina libraries). She collaborates with the University of Milan, University of Brescia, University of Siena. She is also involved in studies and activities about communication and accessibility. She previously led the OTANEXT-AUT project (Interomics Flagship) and the Genetics Unit of the Health Minister GR-09 1570296 project, to investigate the role of mycotoxins in autism onset by a gene-environment interaction perspective. She took part in cancer studies and participated in the PON project VIRTUALAB and in AIRC projects for the discovery of biomarkers of soft tissue sarcomas. As for the agricultural field, she participated in the EU projects DNA-Track and BovMap.
Nadir Yalcin
Dr. Nadir Yalcin works full time as an assistant professor and clinical pharmacist at Hacettepe University, Ankara, Turkey. He is currently taking part in the KU Leuven Pharmacometrics Group as an invited researcher for a short period. His areas of expertise include pharmacotherapy, machine learning, psychopharmacology and nutrition in paediatrics. He currently has more than 30 SCI articles and his H-index is 9.