Invited Speakers

1. Building Bridges

Prof. A Andrea Ballabio

Andrea Ballabio obtained his M.D. degree at the University of Naples, Italy, where he completed his residency in Pediatrics. After working as post-doctoral fellow at the […]

Prof. dr. J.H.J. Jan Hoeijmakers

Jan Hoeijmakers is Prof. Molecular Genetics at the Erasmus Medical Center in Rotterdam, the Netherlands. His research focuses on the mechanism and clinical impact of mammalian […]

Prof. dr. J.C. Hans Clevers

Hans Clevers obtained his MD degree in 1984 and his PhD degree in 1985 from the University Utrecht, the Netherlands. His postdoctoral work (1986-1989) was done […]

2. RNA based therapy and gene therapy

Prof. A Alessandra Biffi

Alessandra Biffi is in charge of the Pediatric Hemathology, Oncology and Stem cell transplant clinics and laboratory at University Hospital of Padua since October 2018 and […]

Prof. dr. A.M. Annemieke Aartsma-Rus

Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. She played an important […]

Prof. A Alberto Auricchio

Alberto Auricchio, MD is Professor of Medical Genetics at the Department of Advanced Biomedicine, “Federico II” University in Naples, and Coordinator of the Molecular Therapy Program […]

3. Regenerative Medicine

Prof. S. Stephen Strom

Prof. G. Graziella Pellegrini

Graziella Pellegrini is Full Professor at the University of Modena and Reggio Emilia and Cell Therapy Program Coordinator at the Centre for Regenerative Medicine "Stefano Ferrari". […]

Prof. dr. C.L. Christine Mummery

Christine Mummery studied physics at the University of Nottingham, UK and has a PhD in Biophysics from the University of London. After positions as postdoc and […]

4. Mitochondrial disorders

Prof. M.J. Marni Falk

Marni J. Falk, M.D., is Executive Director of the Mitochondrial Medicine Frontier Program at The Children's Hospital of Philadelphia (CHOP), Clinical Geneticist, and Associate Professor in […]

Prof. S. Shamima Rahman

Professor Shamima Rahman is Professor of Paediatric Metabolic Medicine at the UCL Great Ormond Street Institute of Child Health (ICH), and honorary consultant at Great Ormond […]

Prof. E.A. Eric Schon

My graduate work in the laboratory of Dr. Jerry Lingrel at the University of Cincinnati (1979-1983) focused on the structure and function of goat globin genes. […]

5. Metabolomics / System biology

Prof. Thomas Hankemeier

Thomas Hankemeier is full professor of Analytical Biosciences, chair of the Division of Systems Biomedicine and Pharmacology at the Leiden Academic Centre for Drug Research (LACDR), […]

Prof. I. Thiele

Professor Ines Thiele is the principal investigator of the Molecular Systems Physiology group at the National University of Ireland, Galway. Her research aims to improve the understanding […]

Dr. C. Carmen Argmann

Dr. Argmann has a doctorate from the faculty of Science at the University of Western Ontario, Canada where she showed that PPARγ and LXR activation could […]

6. Lysosomes: innovative insights

Dr. Simon Jones

Simon Jones is a consultant in paediatric inherited metabolic diseases at the Willink Unit at St. Mary’s Hospital in Manchester, UK. His major research interest is […]

Prof. P. Saftig

Paul Saftig, Biochemical Institute at the University Kiel is director of the department. He is chairing a group of scientists working at projects aiming to understand […]

Dr. M. Huizing

Dr. Marjan Huizing is a basic scientist with a background in cell biology of metabolic disorders. She received a MSc degree in molecular life sciences at […]

7. Invited Parallel Speakers

Prof. dr. Stefan Kölker

A. Personal Statement Prof. Dr. Stefan KÖLKER, MD, is Head of the Division of Child Neurology and Metabolic Medicine at the University Children’s Hospital in Heidelberg, […]

Dr. J. Rho

Dr. Rho is a Professor of Neurosciences and Pediatrics at the University of California, San Diego, and Division Chief of Pediatric Neurology at the Rady Children’s […]

Prof. P. Laforêt

Pascal Laforêt, MD, PhD, is a professor of Neurology at the Versailles-Saint Quentin University, consultant specialized in neuromuscular disorders (myasthenia gravis, muscular dystrophies, and metabolic myopathies) […]

8. Speakers Educational Session

Sjef Smeekens

Sjef Smeekens (ORCID 0000-0001-6631-9426) is professor of Molecular Plant Physiology, Vice-Dean of the Faculty of Sciences and leader of the Master’s programme Science&Business Management at Utrecht […]

Dr. Manuel Goncalves

Manuel Gonçalves received his M.Sc. degree in Plant biotechnology from the University of Lisbon, Portugal. After a post-graduation period in a gene therapy company, he was […]

Prof. dr. R. C. Hoeben

Prof. dr. Hoeben is a graduate of Utrecht University (Biology) and performed his PhD research at Leiden University in the lab. of prof. Van der Eb. […]

9. Speaker Garrod Award Lecture

Prof. Dr. Johannes Häberle

Johannes Häberle is a medical graduate of the University of Tübingen (Germany), and has formal qualifications in pediatrics, neonatology and intensive care. He is a Senior […]

10. Speaker Komrower Lecture

Dr Christine Vianey-Saban

Christine Vianey-Saban is Head of the Department for Inborn Errors of Metabolism and Newborn Screening at the Centre de Biologie et de Pathologie Est in Lyon. […]