Had worked as a journalist and tv presenter focusing on news casting and serving primarily the international desk for over a decade before transitioning to a different professional field as a Marketing and PR Manager for one of the top tier general insurance companies in Cyprus. Nowadays she’s teaching French in public schools promoting the broadening of horizons to students. Since 2006 she has been presenting and coordinating events ranging from conferences to awards.

Prof Leonidas A. Phylactou received his Bachelor’s degree in Medical Biochemistry with honours from the University of Birmingham, UK in 1992 and his PhD in Molecular Genetics/Gene Therapy from the same University in 1995 in collaboration with the University of Connecticut Health Center, USA. Upon completion of his studies, he moved to Oxford University as a post-doctoral scientist where he led a research team on the use of catalytic RNAs as tools for gene therapy in the brain and muscle. Upon returning to Cyprus in 1998 he was employed at The Cyprus Institute of Neurology and Genetics (CING) where he established a research team to investigate and develop novel ways to study gene function and exploit gene therapy approaches. In 2005, he was appointed as the Head of the Department of Molecular Genetics, Function and Therapy, which he still directs.

From 2012 until 2015, Prof. Phylactou served as the first Dean of the newly established post-graduate school of the CING, the School of The Cyprus Institute of Neurology and Genetics.  

From March 2014 to July 2015, Prof. Phylactou served as the Acting CEO and Medical Director of the CING and Acting Provost of the School of the CING. In November 2015, he was appointed as the CEO and Medical Director of the Cyprus Institute of Neurology and Genetics and the Provost of the School.

During his scientific career Prof. Phylactou has secured research funding from several local and international funding organizations and published extensively in the areas of molecular genetics, gene function and gene therapy. He also serves on the Editorial Boards of international peer-reviewed journals.  Prof. Phylactou received the ‘Distinguished Researcher Award 2023’ and the ‘Cyprus Quality Leader of the Year 2024’.

Prof. Leonidas A. Phylactou earned his BSc in Medical Biochemistry (1992) and PhD in Molecular Genetics/Gene Therapy (1995) from the University of Birmingham. After postdoctoral research at Oxford University, he returned to Cyprus in 1998 to join the Cyprus Institute of Neurology and Genetics (CING), where he established a leading research team and later became Head of Department. He served as Dean (2012–2015), Acting CEO/Provost (2014–2015), and CEO/Medical Director and Provost from 2015. He has published extensively, secured major funding, and received the Distinguished Researcher Award 2023 and Cyprus Quality Leader of the Year 2024.

Michael Komodromos is the President of the Cyprus Institute of Neurology and Genetics. He is a Professor of Electrical Engineering and Vice Rector for Research and International Relations at Frederick University. He previously chaired the Electricity Authority of Cyprus and served as Vice Chair of ETYFA. He is a reviewer of journal papers, a member of scientific conference committees, and an evaluator of European programs. He is also an approved evaluator for the Accreditation Board of Engineering and Technology (ABET) in the United States and for the Hellenic Authority of Higher Education. He is a Senior Member of both OSA and IEEE.

Olivér Várhelyi (born 22 March 1972 in Szeged, Hungary) is a Hungarian lawyer and diplomat serving as the European Commissioner for Health and Animal Welfare since December 2024. He holds a law degree from the University of Szeged and a Master in European Legal Studies from Aalborg University. Várhelyi began his career in Hungarian public administration and later worked in Brussels on EU legal and diplomatic affairs. From 2019 to 2024, he was European Commissioner for Neighbourhood and Enlargement. Known for his experience in European law and policy, he has played a significant role in shaping EU external relations

Mr Nicodemos Damianou was born in Lefkosia in 1973. He holds a Sc.B Degree in Computer Science from Brown University USA, a Ph.D in Computer Security & Policy-based Management of enterprise-wide Computer Networks from Imperial College UK, a Diploma of Information Security from Imperial College, and an MBA from the University of Cyprus. He is an IT and Information Security professional with many years of experience in Payment Systems and Fintech, Risk Management Methodologies and Digital Transformation Technologies. He served at managerial positions within the Bank of Cyprus Group. From November 2017 to January 2024 he was the General Manager of JCC Payment Systems Ltd. He is married to Rodoula Damianou and has two daughters, Eleni and Sophia.

Neophytos Charalambides is the Minister of Health of the Republic of Cyprus, having assumed office in December 2025. He holds political responsibility for the formulation, coordination, and oversight of national health policy, with a focus on strengthening public health, improving access to quality healthcare, and enhancing the resilience and sustainability of the health system. His portfolio includes health legislation, public health and prevention, pharmaceutical policy, health workforce policy, and the institutional oversight of key health bodies, including the State Health Services Organisation (OKYpY) and the Health Insurance Organisation (GESY). Prior to his appointment as Minister, Mr Charalambides practised law for over two decades, specialising in personal injury litigation, civil litigation, and criminal law. He is a Barrister-at-Law of the Honourable Society of Gray’s Inn and a member of the Association of Personal Injury Lawyers (UK). His legal career includes extensive courtroom experience, the drafting of complex agreements, and the provision of legal advice to local and international clients. Mr Charalambides has long-standing experience in public life and local government. He served as Deputy Mayor of Limassol Municipality, as an elected member of the Limassol Municipal Council, and in several senior positions within the Democratic Party (DI.KO), including Vice President, a position he holds to date. He has represented Limassol Municipality in European and international fora and has participated in high-level policy dialogue initiatives. He holds a Bachelor’s Degree (LLB) and a Master’s Degree in Law (LLM).

Stella Kyriakides served as the Commissioner for Health and Food Safety of the European Union from December 2019 to November 2024 . She graduated  in psychology from the University of Reading with a master’s degree from the University of Manchester, United Kingdom. She worked as a clinical psychologist in the Mental Health Services of the Ministry of Health in the field of child and adolescent psychiatry. From 2006 to 2019, she served as a Member of the Cyprus Parliament . From 2011-2019 she was head of the Cyprus delegation to the Parliamentary Assembly of the Council of Europe. In 2017, she was elected as the 30th President of the Parliamentary Assembly of the Council of Europe, the fourth woman to hold this position in the history of the Assembly, and the only Cypriot. She founded Europa Donna Cyprus, of which she served as President for 15 years and  was elected President of the European Coalition  for Breast Cancer Europa Donna. She was  appointed, President of the National Cancer Committee of Cyprus to draw up the Cyprus National Cancer Plan. Mrs Kyriakides has received internationally  numerous awards for her work in  patient advocacy. Ms. Kyriakides was awarded an honorary Doctorate from the Department of Sciences of the University of Reading for her overall contribution to the field of health. In 2024 Mrs Kyriakides was awarded the European Cancer League Honorary Award for her work in cancer prevention and treatment across Europe , and  in 2025 received the Yvonne 2025 Lifetime Achievement  Award in Oncology . Mrs Kyriakides currently serves on the Advisory Board of the European School of Oncology, is a Senior lecturer of the school. She is a member of the Board of the University of Cyprus. She has been selected by the World Economic Forum to be a Global  Champions for Womens health.

Dr Milena Angelova has been serving as Secretary-General of the Bulgarian Industrial Capital Association since 2002. SGI Europe Vice-President 2011-2025. She’s been a Member of the European Economic and Social Committee since 2007. Member of the Bulgarian Economic and Social Committee since 2006, and its Vice-President for 2025. In June 2022, Dr Angelova was appointed Bulgarian SME Envoy. She has thirty years of successful track record in representing the interests of the business community at EU and national level regarding important topics such as sustainable development and CSR, SME promotion, innovation and knowledge transfer, skills, future of work, life sciences, etc. Associate professors on Marketing and Sustainable Development at the Economic Research Institute at Bulgarian Academy of Sciences.

François LAMY acting as a vice-president of the AFM-Téléthon.

François Lamy is the father of an 18-year-old boy with Duchenne muscular dystrophy. He has been a board member of AFM-Téléthon (France) since 2012, becoming vice-president in 2017. He is also a board member of the CECS-ISTEM (Stem Cell Research Centre) and chair of the Patient Advisory Board of the EURO-NMD ERN.

AFM-Téléthon was founded by families who realised that their only chance of conquering the disease was to join forces. Starting with just a few families, it has become a key player in the development of treatments for rare diseases, supporting 40 clinical trials in more than 30 pathologies.

Virginie Hivert is Acting CEO and Head of Therapies & Access at EURORDIS–Rare Diseases Europe. She drives the organisation’s strategic work to advance the development of medicines for people living with rare diseases and to embed meaningful patient engagement across research, regulatory processes and access pathways. Since joining EURORDIS in 2014, she has been a leading figure in the organisation’s collaboration with the European Medicines Agency, serving as an Observer on the Committee for Orphan Medicinal Products, as the PRAC Alternate member representing patient organisations, and now as a Civil Society representative on the EMA Management Board. Internationally, Virginie has played an influential role in the International Rare Diseases Research Consortium since 2011, including four years as vice chair of its Therapies Scientific Committee. She brings more than 25 years of experience across research, healthcare and rare disease advocacy, supported by a PharmD and a PhD in Biological Sciences.

Dr. Menelaos Pipis is a Senior Neurology Consultant and Head of the Neuropathology Department at the Cyprus Institute of Neurology and Genetics. He completed his PhD at University College London, specialising in inherited peripheral neuropathies, and holds MRCP qualification from the Royal College of Physicians. He obtained his MBBS from UCL Medical School with distinctions and also earned a First Class Honours BSc in Medical Sciences with Neuroscience. He trained and worked in leading London hospitals, including St Thomas’, Guy’s, King’s College Hospital, and the National Hospital for Neurology and Neurosurgery, progressing to Acting Neurology Consultant. He has received several prestigious academic and national awards recognising excellence in performance, research, teaching, and leadership. His clinical and research work focuses on neuromuscular and neurological disorders, and he has contributed significantly to advancing the diagnosis and understanding of inherited neuropathies.

Tim Leest (born 1982) is Chair of the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency and Senior Clinical Assessor at the Belgian Federal Agency for Medicines and Health Products (FAMHP), a role he has held since 2008. He obtained master’s degrees in Biology and Bioinformatics from the Catholic University of Leuven (KUL). From 2007 to 2024, he served as the Belgian delegate to the COMP and was elected Chair in 2024. He has also been a member of the Management Board of the Fund Rare Diseases of the King Baudouin Foundation and contributes to several European rare disease initiatives.

Donata Meroni is the Head of the Unit ‘Health Monitoring and Cooperation, Health Networks’ within the Public Health Directorate of DG SANTE, European Commission. Her role involves overseeing activities on cross-border healthcare Directive, on rare diseases, and the European Reference Networks for rare and complex diseases.
Previously, she led the unit focused on ‘Health promotion, disease prevention, and funding instruments’ in the same Directorate. From 2017 to 2021, she was the Head of the ‘Health and Food Safety’ Unit at the Executive Agency for Consumers, Health, Agriculture and Food (CHAFEA) in Luxembourg. She has been with the European Commission since 2002, beginning her tenure at DG SANCO, starting her career at the Commission at the Food and Veterinary Office in Ireland

Holm Graessner has graduated in Biomedical Engineering, Cybernetics, Electrical Engineering, German Language and Literature, Philosophy as well as Business Administration. He received his PhD “Summa cum laude” in 2004 and, then, he obtained his MBA degree in 2008.

He has been Managing Director of the Rare Disease Centre, since 2010, at the University and University Hospital Tübingen, Germany. www.zse-tuebingen.de He is Coordinator (Lead PI) of the European Reference Network for Rare Neurological Diseases (ERN-RND, www.ern-rnd.eu). Together with Olaf Riess, he was leading the H2020 Solve-RD project on “Solving the unsolved rare diseases” (www.solve-rd.eu) and also co-leads the Clinical Research Network of European Rare Disease Research Alliance (www.erdera.org) that has started on 1 Sept 2024. He is a founding member of the European 1 Mutation 1 Medicine initiative and member of the Board of Directors of the N-of-1 Collaborative.

In 2020, he has been appointed fellow of the European Academy of Neurology (EAN) and since 2025 he is a Commissioner in the Lancet Commission on Rare Diseases.

In 2023, he received the EURORDIS Black Pearl Award for Leadership and the first Martha and Wilfried Ensinger Award.

Daria Julkowska holds a PhD in molecular biology and completed postdoctoral research in cellular biology at Institut Pasteur (Paris). She also holds an MSc in Research Management (Paris Dauphine) and has extensive training in EU research policy and communication. Active in the rare disease field since 2010, she coordinated the European Joint Programme on Rare Diseases and is currently Scientific Coordinator of ERDERA, a partnership uniting over 180 institutions across 37 countries. She has built large-scale, multi-stakeholder collaborations spanning academia, funders, industry, clinicians, and patients. She chaired a European Commission Expert Group on partnerships and has received major distinctions, including the EURORDIS Black Pearl Award (2020) and the Prix INSERM (2024).

Dr. Violetta Christophidou Anastasiadou is a Consultant Paediatrician and Clinical Geneticist and Head of the Department of Clinical Genetics at Archbishop Makarios III Hospital and the Cyprus Institute of Neurology and Genetics in Nicosia. Born in Famagusta, Cyprus, she studied Medicine at the National and Kapodistrian University of Athens and later obtained a PhD in Medical Genetics with distinction. She specialized in Paediatrics and completed advanced training in Medical Genetics, including Fulbright scholarships at Johns Hopkins University and Children’s National Medical Center in the USA. Since 1994, she has led clinical genetic services in Cyprus, contributing significantly to patient care, research, and education. She has held teaching roles at the University of Cyprus and European institutions and has served on national and European committees on rare diseases, newborn screening, and bioethics

Kaja is a biotechnologist from education. She obtained her degree at the International Faculty of Engineering at the Lodz University of Technology in Poland. She has been working since 2009 in the European Commission, DG SANTE. For seven years she has worked in the GMO field where she was responsible for the authorisation of the GM food and feed in the EU and she has worked closely with the European Food Safety Authority. In 2016, she has moved to the human health division of DG SANTE where she is responsible for the development of the policy and authorisation of medicines for rare diseases, including the EU pharmaceutical reform. She works closely with the European Medicines Agency and the network.

Dr Lederer is Associate Professor and program coordinator at the CING Postgraduate School, and executive board member of the National Committee for Thalassemia and Other Hemoglobinopathies, the Human Variome Project, and the European Paediatric Translational Research Infrastructure (EPTRI). His department performs (i) clinical hematology and population-wide diagnostic services as national molecular reference laboratory and as HCP under ERN EuroBloodNet, including carrier screening, prenatal diagnostics, PGT-M and NIPT, (ii) translational research for the discovery of biomarkers and disease modifiers, (iii) genome editing and advanced therapy development for rare blood disorders, and (iv) development and coordination of patient registries, databases and epidemiology resources, including the world-leading ITHANET Portal and the ClinGen-recognized Hemoglobinopathy Variant Curation Expert Panel.

Priya Singhal, MD, MPH, is Executive Vice President and Head of Development at Biogen, where she leads the company’s pipeline and has contributed more than a decade of scientific leadership. She has guided over a dozen product approvals and more than 150 development programs, advancing treatments for serious and rare diseases. Before Biogen, Dr. Singhal held senior R&D roles at Zafgen Inc., Vertex Pharmaceuticals, and Millennium Pharmaceuticals. She earned her MPH from the Harvard School of Public Health, her MD in Internal Medicine in Mumbai, India, and is an invited speaker and guest faculty lecturer at Harvard Medical School.

Prof Kleopa is a clinician-scientist coordinating multidisciplinary care and cutting-edge therapies for rare neurological and neuromuscular disorders. His translational research has made significant contributions to understanding the mechanisms of neurogenetic, neuroinflammatory and neuromuscular disorders and to the development of innovative gene therapies for inherited neuropathies and leukodystrophies, with results currently being developed for clinical translation in collaboration with industry. His work has been widely published and recognized by numerous prestigious Awards, including the EAN Investigator Award and the National Distinguished Researcher Award. He is actively involved in ERNs. He serves as Board Member in several international professional and patient associations.

Professor Porteus is a physician-scientist who has focused on developing precision genetic engineering strategies to cure hemoglobinopathies and  rare genetic diseases of the blood and immune system. He is the Director of the Stanford Center for Definitive and Curative Medicine and is the President-Elect of the American Society of Gene and Cell Therapy (ASGCT). In addition to his academic work, he has been involved in the founding of several biotechnology companies including CRISPR Therapeutics and Kamau Therapeutic and is a scientific advisor to several more.

Demetris Skourides serves as the Chief Scientist for Research, Innovation and Technology of the Republic of Cyprus, leading the nation’s strategic agenda in research, innovation, and technology. He plays a central role in shaping national policy and contributing to international dialogue on artificial intelligence, digital transformation, and innovation governance. He chairs the National AI Taskforce and the Board of the Research and Innovation Foundation, and participates in international bodies including JRC, ESAF, Future 100, Ignite Cyprus, and PRIMA. He holds academic and executive credentials from Arizona State University, Henley Management College, Columbia, Stanford, MIT, and Harvard, and has held senior leadership positions at Oracle and Amazon Web Services.

Anton Ussi (MSc) is chief executive officer at EATRIS ERIC, the ESFRI European infrastructure for translational medicine. Joining EATRIS in 2010, he was part of the team responsible for the design and statutory incorporation of the infrastructure, whose mission it is to improve the translation of academic innovations into high impact health interventions. In his current role as CEO since 2015, Ussi has a background in technology transfer, with previous history in mechanical engineering and automotive design, and small business administration. Ussi is also Principal Investigator of REMEDi4ALL, a large multi-sector EU- and UK-funded initiative comprising 26 partners.  The REMEDi4ALL mission is to deliver functioning pan-European medicines repurposing eco-system covering all development phases and market entry.

Since 2005 Birutė Tumienė is a member of the Society for Study of Inborn Errors of Metabolism, SSIEM, since 2006 she is one of the founders and a board member of the Baltic Society of Inherited Metabolic Diseases (Baltic Metabolic Group).

She received professional training in traineeships in the Institute for Medical Genetics, University of Ljubljana Medical Centre(Slovenia), Service d’ Endocrinologie, Maladies Osseuses, Génétique et Gynécologie Médicales, CHU de Toulouse (France), National Centre for Inherited Metabolic Disorders, Dublin (Ireland), St Radboud Universitety Hospital, Department of Inherited Metabolic Diseases (the Netherlands), Amsterdam VU Medical Centre, Laboratory for Inherited Metabolic Disorders (the Netherlands), Children Memorial Health Institute, Department of Inherited Metabolic Diseases (Poland), SSIEM Academy Courses in Lisbon (Portugal) and Amsterdam (the Netherlands).

Hélène joined in 2010 the European Commission where she worked firstly in the Directorate-General in charge of health policies (DG SANTE), in particular in the team supporting the establishment of the 24 European Reference Networks (ERNs) on rare diseases. She moved to DG Research & Innovation in January 2020, Unit “Health innovations and ecosystems”, to work on rare disease (RD) research, where she supported the implementation of the European Joint Programme on Rare Diseases (EJP RD) and collaborative research projects to improve diagnostics and therapies for RD patients. She helped preparing, and still support, the European Partnership on Rare Diseases, ERDERA.

Maurizio Scarpa, MD PhD, paediatrician, is the Director of the Regional Coordinating Centre for Rare Diseases at the University Hospital of Udine, Italy. He is Professor of Paediatrics at the Dept. for the Woman and Child Health, University of Padova, Italy, and the Co-Founder of the Brains For Brain Foundation, together with Prof. David Begley, Kings College of London, London, UK. Prof. Scarpa has extensive expertise as a basic scientist in genetics and biotechnology, as well as a clinician in the diagnosis and treatment of paediatric rare disorders; neurometabolic diseases in particular. Together with dr. Christina Lampe he founded the Center for Rare Diseases at the Helios Dr. Horst Schmidt Kliniken in Wiesbaden, Germany.  He is especially interested in developing innovative health approaches for the diagnosis and the treatment of metabolic inherited diseases; to this aim he is also collaborating with major biotech companies as an external independent expert. Prof. Scarpa is the Coordinator of the European Reference Network for Hereditary Metabolic Diseases, MetabERN, formed by 101 healthcare providers in 27 EU countries (www.metab.ern-net.eu ) to facilitate patient-centered holistic activities to implement knowledge, diagnosis, management and treatment for inherited metabolic diseases.

Dr. Giedrė Kvedaravičienė is an expert in health innovation and policy with experience spanning academic, clinical, and governmental settings. Over the last ten years she has contributed to medical innovation through the implementation of advanced therapies, including ATMPs, in clinical practice, while supporting research and policy initiatives at national and EU levels. Her work includes championing population-based and rare disease research with a focus on early diagnosis, translational research, and the integration of innovative diagnostics into health systems. Since early 2024 she serves as Director of Lithuania’s first population biobank at Vilnius University Faculty of Medicine, is a member of the Advisory Boards of the European Partnership for Personalized Medicine (PerMed) and active participant in national and international scientific networks.

He holds a Degree in Medicine and Surgery from the University of Bologna, a PhD in Clinical Genetics from Sapienza University of Rome, and a Master’s in Research Governance from the University of Modena and Reggio Emilia, and was a Visiting Scientist at the US National Cancer Institute. He is Director of the Department of Rare Skeletal Diseases and five National Rare Disease Registries at the Rizzoli Orthopedic Institute, and coordinates ERN BOND, a network of 50 Centers of Excellence across 20 EU countries. He also serves as Delegate to the Italian Ministry of Health for ERN integration. He directs BIOGEN and coordinates major biobank networks, and previously held leadership roles in BBMRI-ERIC and the European Medicines Agency. He leads European research projects on rare diseases and clinical trials. He has authored over 120 scientific publications, served as President of leading international scientific societies, and is a member of several patient organization scientific committees.

Allan Byamukama is the Executive Director  and Founder of the Byamukama Sickle Cell Foundation, where he leads advocacy, education, and support initiatives for individuals and families affected by sickle cell disease. After recently undergoing a successful gene therapy treatment that resulted in healing from sickle cell disease, Allan brings both professional leadership and lived experience to his work. He is deeply committed to improving access to care, advancing awareness, and promoting equity in health outcomes through community-centered and evidence-informed approaches.

Professor Alexis Arzimanoglou, Child Neurologist, is the elected Coordinator of the European Reference Network for Rare and Complex Epilepsies, ERN EpiCARE and Director of the Epilepsy Program at the Barcelona Children’s Hospital San Juan de Dios, Universitat de Barcelona, Spain. Born and studied in Greece, he then worked in the UK and in France.

He has authored/co-authored seven books and over 200 peer reviewed scientific publications. For his global contribution to knowledge generation and epilepsy care he received the ILAE-Epilepsy Ambassador award (2007); the Aicardi award for excellence in Paediatric Neurology from the EPNS ( 2017) and the ILAE-Europe Education award (2022)”.

Professor Nikolaos Zamboglou was born in Limassol, Cyprus. He received his degree in Physics from RWTH Aachen in 1974, followed by a PhD in Physics from the University of Düsseldorf in 1977. He later pursued studies in Medicine, earning his medical degree in 1984 and his PhD in Medicine in 1989 from the University of Essen. In 1992, he was appointed Professor and Director of the Department of Radiation Oncology at the Academic Hospital of Offenbach, Goethe University Frankfurt, a position he held until 2016. Since 1993, he has also served as an affiliated Research Professor at the National Technical University of Athens, within the Institute of Electronic Systems and Computers. From 2003 to 2005, Professor Zamboglou served as President of the German Society of Radiation Oncology. In recognition of his scientific contributions, he was elected Corresponding Member of the Academy of Athens in 2010 and named Honorary Member of the Austrian Society of Radiation Oncology in 2012. In 2015, he received the prestigious Alfred-Breit Prize, the highest honor of the German Society of Radiation Oncology. In 2017, he was awarded an Honorary Doctorate by the Cyprus University of Technology. Professor Zamboglou currently serves as Chief Executive Officer of the German Oncology Center and the German Medical Institute. In 2024, he was awarded the Bundesverdienstkreuz am Bande (Federal Cross of Merit on Ribbon) by the President of the Federal Republic of Germany, one of Germany’s highest civilian distinctions

Dr Anne-Sophie Lapointe has been directly concerned by rare diseases, with two of her children suffering from a lysosomal disease. For 15 years, she has been a member of the boards of directors of national and European associations for rare diseases. She was a member of the INSERM ethics committee for six years.

Anne-Sophie Lapointe has a PhD and a master’s degree in health ethics and economics. Together with the director of Orphanet, she developed some actions in the French National Plan for Rare Diseases 3 (PNMR3), focusing on information, training and e-health. In October 2018, having stepped down from her positions in associations, she joined the Ministry of Health’s rare diseases unit. With the ministry of health, research and industry, she is responsible for steering and coordinating the 75 actions resulting from the PNMR4 launched on 25 February 2025. In this capacity, she sits on the European Commission’s Member States Board for European Reference Networks (ERNs) for rare diseases and coordinates the WP8 on data management for the JARDIN (Joint Action rare Diseases INtegration) joint action funded by the EU4HEALTH programme.

Dr. Leopold is an Assistant Professor of Drug Regulatory Science at the Division of Pharmacoepidemiology & Clinical Pharmacology at the Utrecht Institute for Pharmaceutical Science / WHO Collaborating Centre for Pharmaceutical Policy and Regulation. Her research focuses on evaluating pharmaceutical policies along the drug life cycle with the aim to improve affordable access to medicines. She has a particular interest in understanding European regulatory, HTA and pricing/funding processes within cancer care and for orphan medicinal products and uses various data sources (such as from registries, claims data from insurers, legal and policy documents as well as interview data) in her research.

From 2014 to 2018, Dr. Leopold was a Senior Research Fellow in the Department of Population Medicine at Harvard Medical School and the Harvard Pilgrim Health Care Institute. Prior to that, she worked as a senior researcher at the Austrian Public Health Institute, Department of Health Economics / WHO Collaborating Center for Pharmaceutical Pricing and Reimbursement Policies, where she co-led the Austrian Centre for Rare Diseases as well as the European Network of public authorities of Pharmaceutical Pricing and Reimbursement Information (PPRI).

She holds a doctorate in Pharmaceutical Policy from Utrecht University, Utrecht Institute for Pharmaceutical Science / WHO Collaborating Centre for Pharmaceutical Policy and Regulation, a Master of International Health Care Management, Economics and Politics from Bocconi University (Italy) and a Master of International Business Relations from the University of Applied Science (Austria).

A committed advocate for individuals living with rare diseases, with a strong focus on Duchenne Muscular Dystrophy, actively supporting patients and families through continuous engagement and organizational leadership. My work includes participation in committees addressing patient concerns, helping improve support mechanisms and ensuring that patient voices are heard. I closely follow scientific and medical developments in the field of rare diseases and take part in seminars, workshops, and international conferences to stay informed and contribute to broader awareness. Through involvement in various Cypriot organizations and representation in international bodies, I work to strengthen collaboration and advance the rights and well‑being of patients.

Marina Vasiliou is President and Managing Director of Biogen France, a leading biotechnology company that pioneers innovative science in rare neurodegenerative diseases, immunology, Alzheimer’s disease and neurology. She brings over 20 years of international leadership experience across commercial, marketing, and general management roles in the pharmaceutical industry. She began her career at MSD /Merck in the United States before holding senior positions in France and globally. Marina joined Biogen in 2017 as Vice President and General Manager of Biogen Canada, before assuming leadership of Biogen France in 2021. She holds an MBA from Penn State, is a Fulbright scholar, and earned a Master’s degree in Sociology from EHESS in Paris.

Spyros Polyviou is Director of the Cyprus Alliance for Rare Disorders, where he leads national and European initiatives supporting the advancement of treatments for rare diseases through policy development, patient engagement, and system-level collaboration. He holds an MBA and a degree in Sociology and is a trained patient expert through the EURORDIS Academy in Medicines Research & Development and Scientific Innovation & Translational Research, as well as the EUPATI Patient Experts Programme. He contributes to national health committees and EU-funded projects addressing diagnostic pathways, research translation, and equitable access to innovative therapies for people living with rare diseases.

Harald H.H.W. Schmidt, MD, PhD, PharmD is Professor at Maastricht University and Coordinator of the Horizon Europe platform REPO4EU and the emerging European research infrastructure REPOSYSTEM. Trained in both medicine and pharmacy, he has held academic and leadership positions in Germany, the United States, Australia, and the Netherlands. His work advances drug discovery and drug repurposing to new levels of precision through network and systems medicine, particularly for rare diseases and conditions with unmet medical need, with a strong focus on reimbursement-relevant evidence generation and, consequently, real-world market impact.