Speakers
AIUTI Alessandro
Alessandro Aiuti is M.D., specialized in Immunology, and Ph.D. in Molecular and Cell Biology. In 1998 he obtained the National Board in Hematology. Heis Deputy Director, Clinical Research Coordinator, and Head of Unit on Pathogenesis and Therapy of PIDof the San Raffaele Telethon Institute for Gene Therapy in Milan; Director of the Pediatric Immunohematology Unit, […]
ARDIGÓ Diego
Dr. Ardigò is an MD with a specialization in Internal Medicine. He obtained his PhD at the University of Parma (Italy) and did a post-doctoral fellowship at Stanford University (California, US). Before joining the industry, he worked at the University of Parma (Italy) in the field of cardiovascular and metabolic genomics, and as free-lance consultant […]
ATHANASIOU Dimitrios
Dimitrios ATHANASIOUholds a BA in Business Administration and an MBA in Financial Management. He speaks three European languages and has more than 25 years’ experience with international business projects, working in various countries in consulting, developing and reorganizing companies. When his son was diagnosed with Duchenne Muscular Dystrophy, a fatal and incurable rare disease, he […]
ATIF Uzma
Uzma Atif has more than 20 years’ experience in the Pharmaceutical industry. She joined Shire US Medical Affairs in 2010 and served as an Executive Medical Science Liaison in the area of rare genetic diseases, contributing to and supporting medical and scientific strategy. Since 2018, she has also been a key contributor to the Global […]
BARTOSZEWICZ MORITZ Maja
Raised in a family of doctors, Maja, instead chose a career working across the fields of staff training and sales management. Most recently she was a supervisor and patient advocate within a dementia team in Stockholm. However following the birth of her daughter Ida, she found herself on a different journey – a dedicated search […]
BEDLINGTON Nicola
Nicola Bedlington is Special Advisor at the European Patients’ Forum. Until recently she was Secretary General and joined as its first Executive Director in June 2006. She was the Founding Director of the European Disability Forum, an umbrella organisation uniting over 70 European disability non-governmental organisations (NGOs) to advocate for the human rights and inclusion […]
BEGHYN Terence
Terence is 40 years old, native of Région Hauts-de-France in France. He is pharmacist and holds a Ph.D. in medicinal chemistry. From 2007 to 2014, he is assistant Professor at the Faculty of Pharmacy (University of Lille). Passionate about all drug discovery technologies, he participated in the establishment of several high technology platforms and in […]
BELLGARD Matthew
Professor Matt Bellgard is the inaugural eResearch Director at Queensland University of Technology (QUT). Over his career Professor Bellgard has honed his skills in digital transformation (DT) leadership within and across large institutions addressing cross jurisdictional challenges. He has personally attracted over m in research funding, co-inventor of 5 full/20 provisional patents, designed and commissioned […]
BELMONT John
Dr. John Belmont Senior Principal Medical Scientist, Illumina, Inc., San Diego, California Professor (Adjunct), Department of Molecular and Human Genetics, and Pediatrics, Baylor College of Medicine, Houston, Texas Dr. Belmont is currently a Senior Principal Medical Scientist in the Medical Genomics Research (MGR) team at Illumina, Inc. At Illumina, Dr. Belmont consults on diverse programs […]
BROOKS Philip John (P.J.)
Philip John (P.J.) Brooks is a Program Director in the NCATS Office of Rare Diseases Research (ORDR). Dr. Brooks received his Ph.D. in neurobiology from the University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at the Rockefeller University, Brooks became an investigator in the intramural program of the National Institute on […]
CARTIER-LACAVE Nathalie
MD, Director of the INSERM lab NeuroGenCell (Gene and cell Therapy for neurodegenerative diseases of adults and children) at Institute for Brain and Spine (ICM) in Hospital Pitié Salpêtrière in Paris. Engaged in the development of gene therapy applications for neurodegenerative diseases (genetic leukodystrophies, Huntington’s disease, Alzheimer disease, Amyotrophic Lateral Sclerosis) Including all translation steps […]
CAULFIELD Mark
Mark Caulfield graduated in Medicine in 1984 from the London Hospital Medical College and trained in Clinical Pharmacology at St Bartholomew’s Hospital where he developed a research programme in molecular genetics of hypertension, which has discovered over 1000 gene loci for blood pressure. He served on the NICE Guideline Group for hypertension and was President […]
DJOUDALBAYE Benjamin
Dr Benjamin Djoudalbaye is Head of Policy and Health Diplomacy at the Africa Centers for Disease Control and Prevention. He was a Senior Health Officer for HIV/AIDS, tuberculosis, malaria and other infectious diseases at the African Union Commission from 2009–2018. He has a strong professional experience in strategic planning, administration, management and evaluation of policies, […]
GABALDO Michela
Michela Gabaldo is Head, Alliance Management & Regulatory Affairs at Fondazione Telethon in Milan (Italy) supporting the 2 internal Scientific Institutes SR-TIGET (San Raffaele-Telethon Institute for gene therapy) and TIGEM (Telethon Institute of Genetics and Medicine) through the early development up to the registration and market access of the gene therapy programs developed both internally […]
GAHL William
Dr. William A. Gahl earned his B.S. in biology from the Massachusetts Institute of Technology in 1972 and his M.D. and Ph.D. from the University of Wisconsin. He served as pediatric resident and chief resident at the University of Wisconsin hospitals from 1976-80. In 1984, he completed clinical genetics and clinical biochemical genetics fellowships at […]
HIVERT Virginie
Virginie Hivert joined EURORDIS in 2014 as Therapeutic Development Director. Virginie is responsible for following the development of orphan medicinal products as an observer on the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency. She coordinates the group of high-level EURORDIS representatives/volunteers who sit on the various scientific committees/working parties at the […]
HOUŸEZ François
François Houÿez is working at the European Organisation for Rare Diseases Eurordiswhere he is Director of Treatment Information and Access. He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases since 2003. He pioneered patient advocacy with the European Medicines Agency as part […]
JONKER Anneliene
Anneliene Jonker, PhD, is the Funding and Strategy Officer of the TechMed Centre, at the University of Twente, in Enschede, The Netherlands. In this position, she is responsible for setting up funding strategies for different researchers in the personalized medicine-, medtech, and rare diseases domain, working with academic and industrial researchers. In addition she assists […]
JULKOWSKA Daria
Daria Julkowska has over 15 years of experience in research and management. She is the Scientific Coordinator of the European Joint Programme on Rare Diseases that brings together different type of stakeholders (researchers, funders, clinicians & patients) from 35 countries from Europe and beyond, and also is responsible for the coordination of the IRDiRC Scientific Secretariat. This […]
KRAWITZ Peter
Dr. Peter Krawitz became FDNA’s Chief Science Officer with the goals of identifying and exploring high-impact and original research fields to further the development and use of NGP technologies, as well as driving the research efforts to create new knowledge that serves to expand the field of genetics. Beyond his role at FDNA, Dr. Krawitz also serves […]
LE CAM Yann
Yann was one of the founders of EURORDIS-Rare Diseases Europe in 1997. He is the organisation’s Chief Executive Officer since 2000. Yann initiated Rare Diseases International (RDI) in 2009. He is an elected member of the RDI Council and Chair of the RDI Advocacy Committee. He is a founding member of the NGO Committee for Rare […]
MENZEL Olivier
Dr. Olivier Menzel graduated (B.Sc.) from the University of Geneva, where he obtained a Master of Medical Genetics (M.Sc.) in 2001 and a Ph.D. in Molecular and Cellular Biology in 2006 from the University of Lausanne and EPFL at the Swiss Institute for Experimental Cancer Research (ISREC). For seven years, he directed the laboratory of […]
MONACO Lucia
Lucia Monaco is the current Consortium Assembly Chair of the International Rare Diseases Research Consortium (IRDiRC), where she represents Fondazione Telethon, the Italian charity committed to rare genetic diseases research. She is the head of Research Impact and Strategic Analysis and former Chief Scientific Officer of Fondazione Telethon in Milan, Italy. She previously worked as […]
NELLÅKER Christoffer
Christoffer Nellaker is a researcher in Digital Phenotyping based at the Big Data Institute at the University of Oxford. His research encompasses broadly extracting biologically meaningful data from imaging modalities. Dr. Nellåker’s group is translating the latest developments in computer vision and computational biology to aid diagnosis of rare diseases. The work is a collaborative […]
NORSTEDT Irene
Irene Norstedt works at the European Commission, where she is Acting Director responsible for the Health Directorate within the DG for Research and Innovation, European Commission. She is also Head of the Innovative and Personalised Medicine Unit. She has been at the European Commission since 1996, and was instrumental in the creation of IMI in […]
OLLENDORF Daniel
Dan Ollendorf is Director of Value Measurement and Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts University. His research interests include expanding the use of health technology assessment in low- and middle-income economies, as well as refinement of value assessment tools in the US and […]
PARISER Anne R.
Director, Office of Rare Diseases Research National Center for Advancing Translational Sciences (NCATS) National Institutes of Health (NIH) Anne Pariser, M.D. is the director of the Office of Rare Diseases Research (ORDR) at the National Center for Advancing Translational Sciences (NCATS) NIH. ORDR is dedicated to accelerating rare diseases research to benefit patients, through rare diseases […]
PEARCE David A.
David Pearce is President of Innovation and Research for Sanford Health. He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986. He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK. […]
REYMOND Alexandre
Alexandre Reymond carried out his thesis in the laboratory of Dr. Viesturs Simanis at the Swiss Institute for Experimental Cancer Research (ISREC) and received his Ph.D. from the University of Lausanne in 1993. After completion of his postdoctoral training with Dr Roger Brent in the Department of Molecular Biology, Massachusetts General Hospital and in the […]
SCHEEREN Joseph
Dr. Scheeren started his pharmaceutical industry career in 1982 with Servier in Paris, responsible for Regulatory Affairs Northern and Eastern Europe, and Clinical Development in Munich from 1986 – 1987. In 1991, he was appointed Head of Worldwide Regulatory Affairs at Serono, Geneva. In 1992, he took over responsibility of the Global Regulatory Affairs department […]
SEYFERT-MARGOLIS Vicki
Vicki Seyfert-Margolis, CEO and Founder of My Own Med, Inc., has published and presented in top journals and numerous conferences about the transformative nature of digital technologies and novel clinical trial designs. Prior to founding My Own Med, Inc., Vicki was appointed in the Obama Administration as the Senior Advisor for Science Innovation and Policy […]
SIREAU Nick
Dr Nicolas Sireau is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. He is also co-founder and Chair of Findacure, an organisation that helps rare disease patient groups. Previously, Nick was the CEO of […]
VAN DER GOOT Gisou
Gisou van der Goot is the Head of the Laboratory of Cell and Membrane Biology at EPFL (Ecole Polytechnique Fédérale de Lausanne) in the School of Life Sciences of which she is the Dean since 2014. Before joining EPFL, she was Group Leader at the Faculty of Sciences of the University of Geneva (UNIGE) and […]
VAN KARNEBEEK Clara
Professor Clara van Karnebeek is head of the metabolic diseases department in the Radboud University Medical Centre in Nijmegen and principal investigator at the Amsterdam University Medical Centres, The Netherlands and the University of British Columbia in Vancouver Canada. Clara’s work as a pediatrician and biochemical geneticist focuses on early diagnosis and innovative treatment of […]
VOLK Hans-Dieter
Hans-Dieter Volk, MD, is Professor of Immunology and head of the both Institute of Medical Immunology, Charité Berlin and BIH Center for Regenerative Therapies (BCRT) as well as deputy spokesman of the Berlin-Brandenburg School for Regenerative Therapies (BSRT) (all Berlin, Germany). In addition, he is scientific head of the division Immunology of the Labor Berlin […]
WARD Susan J
Recognizing the urgency of finding new approaches to clinical trial design and analysis in rare disease, in 2015 Dr. Ward founded the collaborative Trajectory Analysis Project (cTAP), a dynamic pan-stakeholder alliance applying advanced data science to the largest collection of patient clinical data in Duchenne Muscular Dystrophy http://ctap-duchenne.org A pharmaceutical executive, scientist, consultant and educator […]
WEDELL Anna
Anna Wedell, MD, PhD, is a Clinical Geneticist and Professor of Medical Genetics at the Department of Molecular Medicine & Surgery and Science for Life Laboratory (SciLifeLab), Karolinska Institutet, and Head of the Centre for Inherited Metabolic Diseases (CMMS) at Karolinska University Hospital. Her multidisciplinary team provides nationwide diagnostics, including newborn screening, as well as […]
WEISSBAECKER Susanne
As Global Head of Access to Medicines Susanne is leading Takeda’s strategy to enhance access and strengthen evolving healthcare systems with a focus on low-and middle-income countries. Prior to this role, Susanne was Head of Patient Access and Services in Takeda Europe and Canada and successfully developed and implemented patient engagement programmes for Entyvio and […]
