Don is the Director for Medicines Development at EATRIS and Scientific Lead of the REMEDi4ALL European Platform for Medicines Repurposing. Don previously headed therapeutic development at the National Center for Advancing Translational Sciences at the US National Institutes of Health following a 30-year career in academia, biotech, non-disease research foundations and patient care organisations.

Anton Ussi has been the EATRIS Operations & Finance Director since 2015. Joining EATRIS in 2010, he was co-responsible for the operational design and statutory incorporation of the infrastructure. With a background in mechanical engineering in the automotive industry, small business administration, and later in technology transfer focused on molecular imaging, Ussi specialises in public private collaboration and deployment of infrastructure for translational research in medicine.

Mary Rose (MayRo) Roberts is the Chief Operating Officer at Beacon: for rare diseases, a UK-based charity building a united rare disease community with patient groups at its heart.
At Beacon, MayRo plays a central leadership role in shaping the organisation’s strategy, operations, and programme delivery. Since joining the charity in 2016 as its first Events Officer, she has led the growth and development of Beacon’s major activities, including patient group training programmes, the Rare Disease Showcase series, and drug repurposing conferences. She now manages a team of six in delivering Beacon’s core projects, oversees key internal operations, and ensures the smooth day-to-day running of the organisation.
Before beginning her career in the small charity sector, MayRo graduated from Keele University with a First Class Honours degree in Politics.

Miikka Vikkula, MD, PhD, is an internationally recognised leader in human and medical genetics, with more than 30 years of research at the intersection of molecular genetics, vascular biology, and rare diseases. He trained in molecular genetics at the University of Helsinki (MD–PhD), completed postdoctoral research at Harvard Medical School, and later earned a second PhD at UCLouvain. In 1997, he founded his independent research group at the de Duve Institute in Brussels, where he has been a Full Member since 2004 and Full Professor of Human Genetics at UCLouvain since 2013.

Professor Vikkula is a pioneer in uncovering the genetic and molecular bases of vascular and lymphatic anomalies. His discoveries identified causes of numerous inherited and sporadic vascular disorders, defined key disease entities, and clarified central pathogenic pathways, including PI3K–AKT–mTOR and RAS–MAPK signaling. A defining feature of his work is its clinical translation: his team developed disease models and contributed to multiple clinical trials, including the phase III VASE trial of sirolimus and precision-medicine studies for arteriovenous malformations. 

He has authored over 250 research articles, cited more than 31,000 times (H-index 88), and received major international awards. He is a member of the Royal Academy of Medicine of Belgium and a Commander of the Order of Leopold. 

Dr Cesar Hernández García is Director General of Common Portfolio of the NHS and Pharmacy at the Spanish Ministry of Health since 2022. Formerly, he was Head of Department of Medicines for Human Use at the Spanish Agency for Medicines and Medical Devices from 2009 to 2022. A rheumatologist by training, he worked at the Hospital Clínico San Carlos (Madrid) from 1989 to 2009.

Dr. Yoana Nuevo-Ordóñez, who holds a PhD in Analytical Chemistry, is Head of the Innovation Office and the National Scientific Advice Unit at the Spanish Agency of Medicines and Medical Devices (AEMPS). She represents Spain in the European Innovation Network (EU-IN), which brings together the Innovation Offices of National Competent Authorities and EMA’s Innovation Task Force. Within this network, she has led and co-led major European initiatives, including the STARS project funded by the European Commission, and several Horizon Scanning reports on emerging technologies such as genome editing, faecal microbiota transplantation, and nanomedicines. She also co-leads the Simultaneous National Scientific Advice (SNSA) Pilot with the Paul Ehrlich Institute (PEI) and Belgium’s FAMHP.

In the field of drug repurposing, Dr. Nuevo-Ordóñez co-led the European Repurposing Project Pilot in collaboration with EMA, recently completed, and serves as a member of the REPO4EU expert board while collaborating in REMEDi4ALL. More broadly, she drives the strategic theme “Regulatory Science, Innovation and Competitiveness” within the European Medicines Agencies Network Strategy (EMANS) 2028, promoting regulatory innovation and competitiveness across Europe.

Julián Isla is a software engineer by training and works as a Resource Manager for the Artificial Intelligence group at Microsoft. He has 29 years of experience working for international IT companies, helping customers with digital transformation and leveraging the capacity of artificial intelligence to move organizations into automation. He is also the co-founder of Foundation 29, a non-profit organization focused on how artificial intelligence can empower people to make decisions about their own health, based on the evidence provided by data and supported by decision support systems. Julián is the father of Sergio, a sixteen-year-old boy who has Dravet Syndrome. He founded the Dravet Syndrome Foundation in Spain and the European Dravet Syndrome Federation, where he holds the position of Regulatory Advisor. Despite not having a neuroscience or medical background, he gained the skills to be a member of the Orphan Drug Committee at the European Medicines Agency (EMA) as a patient representative. Julián is also part of the Therapeutic Advisory Group for Eurordis, the largest organisation of rare diseases in Europe. In Spain, he is a member of Ciberer (the Spanish Network for research on rare diseases) scientific advisory group, a member of the advisory board of several hospital research institutions, and a member of the board of five foundations working on healthcare. Julián holds a great combination of skills, combining technology, medicine, science, clinical processes, patient engagement, and regulation.

Dr. Marta Puyol serves as the Scientific Director of the Spanish Association Against Cancer (AECC) and Deputy General Director of the AECC Scientific Foundation, where she shapes and drives the organisation’s national research strategy. She leads the design and execution of programs that fund high-impact cancer research, strengthen Spain’s scientific talent, and elevate the international visibility of Spanish researchers.

She has spearheaded major strategic initiatives, including the creation of World Cancer Research Day, influential national reports on the state of cancer research, international collaborations to increase Spain’s presence in European research networks, and the establishment of AECC’s patient advocacy program, integrating patient perspectives into research and policy. Her work centers on building a more competitive, collaborative, and patient-driven cancer research ecosystem.

Shirlene Badger is passionate about the role of genomics in improving health and leads Global Patient Advocacy at Illumina Inc. In this role she leads the Patient Advocacy strategy and engagement across the fields of rare and undiagnosed genetic disease, reproductive health, newborn screening, infectious disease and oncology. Trained as a medical sociologist at the University of Cambridge, her PhD was one of the first to explore the experiences of children and families participating in genetic research to find a cause for their symptoms. She then spent over fifteen years leading academic research initiatives at the University of Cambridge, UK and elsewhere that sought to elevate the impact of patient voice in the development and implementation of novel and experimental medical and omic technologies.

Dan’s eldest daughter Amelie was diagnosed with Tay-Sachs in 2011 at 15 months of age and, upon finding that there was no dedicated advocacy group providing support for families affected by this disease in the UK, he set set-up the Cure & Action for Tay-Sachs (CATS) Foundation with his wife Patricia. Since the group was launched, they have been able to grow The CATS Foundation so that it now offers a variety of services to its family members and has been actively involved in the research investigating therapeutic treatments for Tay-Sachs and Sandhoff disease. Dan is fully committed to leading the charity towards its goal of finding a treatment so that there is hope for those families affected by Tay-Sachs and Sandhoff in the future.

Dr Nafeesa Mat Ali is a Clinical Researcher at City St George’s, University of London, working at the intersection of clinical trials, diagnostics, and global health. Trained as a medical doctor in Malaysia, she transitioned into research to advance equitable access to affordable treatments and early diagnosis, particularly for underserved populations. 

She is a part of the International Affordable Diagnostics and Therapeutics Alliance (IA-DATA) and collaborates closely with the Universiti Malaya Affordable Diagnostics and Therapeutics (UMADT) team to develop scalable, cost-effective healthcare solutions. Her current work centres on drug repurposing and innovative diagnostics for neglected cancers and rare diseases, including managing international multi-site studies such as the NeoART cancer drug repurposing programme in Malaysia. 

Nafeesa also champions health literacy and community engagement for breast and cervical cancer, co-designing cancer awareness initiatives with ethnic minority women community champions in the UK and indigenous women community champions in Sarawak, Malaysia to make health research more inclusive and impactful. 

Prof. Surrallés is currently the Scientific Director of Sant Pau Hospital Biomedical Research Institute, with over >1300 researchers and 72 research teams. He is also Director of the Joint Research Unit in Genomic Medicine UAB-Sant Pau, and Coordinating Director of the Master of Medical Genetics at the UAB, a join collaboration between Sant Pau and Vall Hebron UAB Hospitals.  

With a PhD in Genetics and postdoctoral experience in The Netherlands (Leiden University Medical Center) and Finland (Finnish Institute of Occupational Health), he originally set up his research team at the UAB where he is currently Full Professor of Genetics. He was member of the University Research Commission and Director of the UAB Department of Genetics and Microbiology. In January 2017, he was appointed Director of the Genetics Department at Sant Pau Hospital, Barcelona. He is Director of the Biobank of DNA Repair Syndromes and team leader at the Centre for Biomedical Network Research on Rare Diseases (CIBERER), where he acted as member of its Direction Board for 10 years (2007-2016).  

He is specialist in the field of cancer predisposition syndromes due to mutations in DNA repair genes, where he has contributed with the discovery of novel causative genes in breast and colon cancer, Fanconi anemia, Bloom syndrome and others. Regarding therapeutic research, he holds two orphan drug designations by EMA, participates in academic clinical research including gene therapy clinical trials, and coordinates a trial on the prognostic value of genetic factors in Fanconi anemia and the first ever clinical trial on Fanconi anemia head and neck cancer.  

Thanks to his extensive research activity, he holds the ICREA-Academia award with four consecutive renewals (2008, 2013, 2018 and 2023). Dr. Surrallés has supervised over 60 research grants as Principal Investigator awarded from public and private institutions worldwide including Catalan, Spanish, European, American and Australian institutions, adding up to >9M euros of competitive funding in the last 15 years.  

Prof. Surrallés is inventor of 5 patents, leads a number of contracts with private foundations and biotech-pharma companies including Moderna, Rocket, Roche, Boehringer Ingelheim, Genzyme, Merck and others. He has given tens of invited lectures in international meetings world-wide and published >150 publications including articles in Nature, Lancet, Nature Communications, Nature Medicine, Cell Stem Cells, Blood, Gastroenterology, Am J Hum Genet, Genes and Dev and PNAS (12.000 citations, H factor of 55). He had supervised 20 PhD students and tens of Master Degree Students.  

He is reviewer of tens of scientific journals including Science, Nature, Molecular Cell Biology or Blood and Referee of multiple national and international funding agencies including European Research Council (EU), Agence Nationale de la Recherche-ANR (France), Agència de Gestió dAjuts Universitarisi de Recerca (AGAUR), ANEP (Ministerio de Ciencia y Tecnología), Cancer Research UK (UK), Commission of the European Union, Selection Board Subcommittee on Genetic Diseases and Cancer of the ISCIII (Spanish Ministry of Science and Innovation), Dutch Cancer Society (The Netherlands), Fanconi Cancer Fundation (USA), German-Israeli Fundation for Scientic Research and Development (Germany), INSERM (France), and the French National Cancer Institute (France). 

Guillaume is an MD, PhD and Professor of Medicine at Hôpital Necker–Enfants Malades, Université Paris Cité (Paris, France). Trained in nephrology and kidney transplantation, he also completed a PhD in molecular and cellular biology and a postdoctoral fellowship at Harvard Medical School. He became Full Professor in 2019. His early research on PI3K/AKT/mTOR signalling in kidney disease, supported by multiple European Research Council grants (Starting, Proof-of-Concept, and Consolidator), laid the groundwork for his later breakthroughs in rare diseases.

In 2018, he made a paradigm-shifting discovery in PIK3CA-related overgrowth spectrum (PROS), a group of severe vascular disorders caused by somatic activating PIK3CA mutations. These conditions lead to disfiguring overgrowth, complex vascular malformations, chronic pain, thrombosis, bleeding, and reduced life expectancy, with previously only symptomatic treatment options. Guillaume and his team identified alpelisib (BYL719), a PI3Kα inhibitor developed for oncology, as a targeted therapy. They generated the first faithful mouse model of PROS, demonstrated reversal of disease manifestations, and conducted a proof-of-concept study in 19 patients showing significant clinical improvement. Published in Nature (2018), this work led to the international EPIK-P1 trial and to U.S. FDA approval of alpelisib in 2022—the first approved therapy for PROS.

He has since extended this precision-medicine approach to other vascular anomalies, including KRAS-mutant arteriovenous malformations treated with sotorasib. Recognized internationally, he has received major scientific awards, secured over €28 million in funding, and delivered more than 150 invited lectures worldwide.

Barbara Yu is Co-founder and President of the Citrin Foundation, a research-driven, patient-centered nonprofit dedicated to finding a cure for citrin deficiency, a rare inherited metabolic and urea cycle disorder, and advancing innovation in the rare disease field. She established the Foundation in 2016 with her husband, Yen How Tai, driven by a family’s experience with this condition and a conviction that meaningful progress in rare diseases requires long-term commitment, cross-sector collaboration, and scientific excellence.

Drawing on a background that bridges science-driven philanthropy, patient advocacy, and finance, Barbara brings a holistic perspective to the rare disease field, addressing both the scientific challenges of therapy development and the systemic barriers that often slow research, diagnosis, and patient access to treatment. She advocates for an ecosystem approach to patient-driven research and philanthropy-led science as a way to accelerate therapeutic innovation in underserved disease areas.

Under her leadership, the Citrin Foundation has built a global research and clinical ecosystem to advance the understanding, diagnosis, and treatment of citrin deficiency. The Foundation supports and leads initiatives spanning basic science, translational research, therapeutic development, clinical studies, patient support, and community engagement, working closely with academic institutions, clinicians, researchers, biotechnology partners, and patient communities worldwide. A key strategic milestone for the Foundation is the establishment of the UCD Translational Research Center Universität Zürich – Citrin Foundation, launched in 2025 to accelerate translational research in urea cycle disorders.

In 2024, Barbara convened and hosted a multi-stakeholder strategy roundtable at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), bringing together regulators, researchers, clinicians, and patient advocates to discuss the essential components required to successfully develop therapies for rare diseases.

In parallel with her philanthropic work, Barbara is Co-founder and Co-CEO of YH2 Capital, a long-term investment firm managing proprietary capital with a fundamental investment approach. Earlier in her career, she was Partner and Head of Asia at Eton Park Capital Management and previously led the Principal Strategies Group in Asia at Goldman Sachs, overseeing the investment business at both firms. She began her career in mergers, acquisitions, and restructurings at Morgan Stanley in New York and Hong Kong.

Barbara graduated from Magdalene College, Cambridge University, with a BA in Law, double first-class honors. Barbara was awarded the N. Diaz Scholarship for Law and Norton Rose Prize for Commercial Law. She was also a Jardine Scholar which provided a full scholarship for her undergraduate study.

Dr. Roman Fudim trained in physics, biophysics, and structural biology at Humboldt Universität zu Berlin and Stockholm University, focusing on GPCRs and SLCs. 

His work spans molecular design through to translational strategy. He contributed to the preclinical development of non-conventional antibody formats within TCE and ADC programs at Morphosys and lead the portfolio strategy at a de novo antibody design startup conceptualising the internal pipeline from target identification to in vivo models.

At biotx.ai, he leads the efforts on translational science and strategic partnerships, supporting programs by utilising human genetics to identify new targets and repurposing opportunities.

Dunja joined ZonMw in 2020 and leads the national Drug Repurposing Programme. She oversees programme operations and drives its strategic development with the Dutch Ministry of Health and other key stakeholders. Her focus is on strengthening the programme’s impact through cross‑sector collaboration, improved knowledge exchange and evidence‑based pathways that accelerate translation into clinical and societal benefit. She also contributes to the European REMEDi4ALL initiative, helping to build a coordinated ecosystem for drug repurposing and advance innovative funding approaches.

Before joining ZonMw, Dunja built a solid 20‑year track record in a wide range of R&D roles within the pharmaceutical and CRO sectors. She contributed to clinical trial start‑up, steered complex multinational projects, and held several leadership positions across the Nordic and Benelux regions.

As work package leader in the EU-funded project REMEDi4ALL Heleen is leading a global funders network and ‘think tank’ for repurposing. This aims to discuss policy issues, share best funding practices, co-ordinate (new) funding streams, promote joint calls and develop innovative co-funding models for drug repurposing, ultimately enhancing the repurposing ecosystem for researchers across Europe. Heleen is a pharmacist by training and holds a PhD in drug utilisation research in older people. She has worked as a pharmacotherapy advisor at the Dutch HTA organisation for a couple of years before she joined ZonMw in 2022. ZonMw programmes and funds research and innovation in health, healthcare and well-being, encourages the use of this knowledge and highlights knowledge needs.

Sibren van den Berg is the director of RARE-NL, a national platform that aims to accelerate the development and accessibility of therapies for rare diseases and through drug repurposing. His work focuses on strengthening the Dutch drug repurposing landscape by connecting academic, clinical, and regulatory stakeholders and actively advancing concrete repurposing cases. In addition, his PhD research at Amsterdam UMC focuses on the availability of drugs for rare diseases, with a particular emphasis on drug repurposing.

Saco de Visser is the scientific director of FAST (Centre for Future Affordable and Sustainable Therapy development). He is a pharmacochemist (VU Amsterdam), registered clinical pharmacologist and holds a PhD on A Question Based Approach to Drug Development (medicine, Leiden University). He has 12 years of experience in managing, financing and conducting international clinical drug research at the Centre for Human Drug Research, ZonMw and Nycomed (now Takeda). He is the initiator of the ZonMw funding programme for rational pharmacotherapy, which he further developed together with Benien Vingerhoed. From 2014 onwards, he combined his position at ZonMw with positions as Director of Cluster Development at Leiden Bio Science Park, Head of Education at Paul Janssen Futurelab Leiden (LUMC) and advisor at Platform Medicine and Society (AmsterdamUMC). He was the initiator and currently scientific director of FAST. Saco is a member of various national and international committees and working groups that advise government, public and/or private parties, on drug development and/or financing.

Alisa DeGrave is a postdoctoral researcher at the Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP-TNM) in Göttingen, working under the mentorship of Prof. Dr. med. Lars Schlotawa. Her research focuses on the molecular mechanisms underlying multiple sulfatase deficiency (MSD) and how repurposed small molecules modulate cellular pathways involved in its pathophysiology. By studying these effects in cell models, she aims to understand how these small molecules rescue the disease phenotype and to identify therapeutically relevant candidates.

Yannick bridges a strong interdisciplinary background in pharmaceuticals and business. With interest spanning from business operations, commercial strategies, partnerships, and innovation in the life sciences sector, he focuses on building collaborations that assist the development of precision medicine. He combines scientific understanding with commercial insight to translate precision medicine capabilities into impactful partnerships across biotech and pharma.

Martin trained as an Engineer and gained his degrees from Oxford Brookes University in 1983 and 1993 respectively. After five years as an Engineering Officer in the British Army, he has held various positions within the ICT sector working for Dell Computers, British Telecom, Novell, Panasonic and Capita Professional Services. He has worked at Dendrite Clinical Systems for 15 years liaising with MedTech companies, as well as national and international medical societies and rare disease organisations, helping them to design and develop bespoke clinical registries to collect data for analyses aimed at improving their outcomes.

Ayna Baladi Nejad is a drug developer with almost a decade of experience as a Clinical Pharmacologist in the pharmaceutical industry, currently biotech. Previously, she served as function leader on Global Project Teams at Novo Nordisk, driving the global programs for priority assets. She has extensive knowledge of regulatory requirements and commercial considerations from preclinical to post-market phases of drug development, and the decision-making to expand to other indications. Prior to her roles in the biopharma industry, she held academic positions in Denmark, US, and France. She holds a PhD in Health and Medical Sciences from the University of Copenhagen. As an ultra-rare disease parent, she independently advocates for access to off-label treatments that are supported by mechanistic evidence and privately sponsors n-of-1 research with collaborators in Germany and UK.

Dominik Cysewski, PhD, is Scientific Director of the PACS2 Research Foundation and an academic researcher at the Medical University of Bialystok, where he leads the Laboratory of Molecular Medicine and Rare Diseases. He leads translational molecular research in PACS2 syndrome, combining patient-derived and animal models, multi-omic profiling, and phenotypic screening to define disease mechanisms and prioritise therapeutic directions. Within the PACS2 programme, he helps coordinate research strategy across patient families, clinicians, academic groups, CRO partners, and international collaborators, including communication between teams and the development of joint grant-funded projects. His current work spans patient-derived cellular models and a genetic PACS2 mouse model, with a focus on turning molecular data into tractable next experiments. More broadly, he is interested in how patient organisations can act not only as advocates or data providers, but as active scientific partners in rare disease research.

Nathalie, PharmD, is a senior scientific project manager at the Innovative Health Initiative (IHI) within the Scientific Operations team, with responsibilities ranging from coordination of activities within the team, support to public-private consortia, to engagement with stakeholders with particular focus on regulatory bodies. Nathalie joined in 2012 the Innovative Medicines Initiative (now IHI), after having worked at the European Medicines Agency (EMA) over 15 years as a scientific administrator with various responsibilities related to the development and approval of medicinal products.

Dr Felix Chan is an Assistant Professor in Pharmacology at University of Birmingham. Dr. Chan leads a research group, Chan Lab (www.chanlab.co.uk) which focuses on brain metabolism and mitochondrial function in rare neurological conditions. Dr. Chan is recognized as a world-leader in rare epilepsy syndromes like tuberous sclerosis complex (TSC) and mitochondrial disease. Dr. Chan’s research work is translational, spanning molecular and cellular discovery to therapeutic development, clinical testing, and patient outcome measurements. Dr. Chan is an avid believer in drug repurposing and its potential to achieve rapid impact for clinical and patient benefit. Dr. Chan also teaches actively in the M.Pharm course program at University of Birmingham and other postgraduate course programs like M.Res in Neurogenetics and M.Sc in Biomedical Sciences. Dr. Chan is a passionate mentor of early career researchers and support trainees from diverse background. Dr. Chan also serves as trustee for HOPE for Epilepsy and scientific advisory board member for UKRET, CureDHDDS, and CRELD1 Warriors.

Dr. Ivo van der Bijl is a pharmacist and cell biologist, holding a PhD from Sanquin Research. He joined Tiofarma B.V. in 2019, starting in regulatory affairs before moving into project management, where he combines scientific expertise with strategic coordination. He has contributed to a range of repurposing projects, including the LoDoCo2 project—a collaborative effort involving multiple companies and cardiologists to study colchicine therapy in patients with chronic coronary disease, and which ultimately led to the submission and approval of the registration dossier.

Nadiah Hanim Abdul Latif is a sustainability and corporate affairs leader with over two decades of cross-sector experience spanning corporate, NGO, humanitarian, social enterprise, academic, and ministry-linked advisory roles. She works at the intersection of strategy, advocacy, and public health, applying her expertise to rare diseases, child and disability rights, and inclusion — strengthening institutions and enabling innovation ecosystems that deliver practical impact.

She serves as President of the Malaysian Rare Disorders Society, is a Board Member of Rare Diseases International, serves as the SEA, South Korea, and MENA regional representative for the Phelan McDermid Syndrome Foundation, and Co-Chairs the Global Rare Disease Network Working Group on Care Pathways. Her work has contributed to landmark efforts, from co-establishing Malaysia’s first reporting portal to combat child sexual exploitation and abuse materials, to advancing national action plans on business and human rights, multi-stakeholder policy platforms, and coordinated care dialogue at national and international levels.

Beyond rare disease leadership, she serves as an Adjunct in academia, a Media Anchor, a socialpreneur, an Assistant Child Protector, and a Child Court Advisor for the Petaling District within the justice system.

Across these roles, she builds ecosystems, connecting diverse stakeholders with the aim to advance coordinated solutions in complex environments.

Nadiah believes meaningful progress happens when we listen deeply, work collectively, and act with both courage and kindness.

Alessandra is the vice president of OIFE (Osteogenesis Imperfecta Federation Europe) and a volunteer for As.It.O.I. (the Italian Association for OI) and for Brittle Bone South Africa.

She has a BA Hons degree in Economics and has worked over 20 years in Corporate with an expertise in team leadership and supervision, a strong foundation in project management, stakeholder engagement and strategic planning. In 2016 she established her own company in South Africa which she still runs.

Alessandra was diagnosed with OI as an adult and since then has developed an interest in learning more about it and being involved with her community.

She has attended EURORDIS Open Academy of Medicine Research and Development and The Open Academy of Scientific Innovation and Translational Research, and she is currently attending the Open Academy ERDERA School in the Data, Ethics and AI training.

She is an EUPATI fellow (cohort 7) and EUPATI HTAR Ambassador, for which she did a training that entitles her to be consulted for JCAs and JSCs at EU level.

She is part of the European Regional Task Force on Rare Diseases that is currently working in the development of the European Blueprint for Rare Diseases, which will lay the foundation for a future EU Action Plan and contribute to the Global Action Plan on rare Diseases.

She is part of EURORDIS Mental Health Network promoting this topic within the OI community by organizing workshops and panels.

She is also a PAG member in an undergoing clinical trial (MOI-A) within the REMEDi4ALL network and she is part of the RealiseD project, a European Innovative Health Initiative (IHI) research program aiming to transform clinical trials for rare and ultra-rare diseases by developing comprehensive methodological and operational frameworks that improve how studies are designed, conducted, analyzed, and implemented.

Alessandra is a dedicated patient advocate that works to increase visibility for underrepresented patient communities, not only in Europe, but also in Africa, with a strong emphasis on empathy and proactive engagement to create an inclusive environment that empowers patients.

Matthew Baker is a patient advocate in the UK living with a rare long sleep disorder called Idiopathic Hypersomnia (IH). He advocates for the condition within both the rare disease and sleep communities, as IH is significantly under‑supported and lacks research compared with its better‑known counterpart, narcolepsy.

Idiopathic Hypersomnia commonly presents with excessive daytime sleepiness and sleep drunkenness, severely limiting quality of life, productivity, and safety. While existing therapeutics do not adequately address these symptoms, case reports suggest that an existing medication may target an underlying neurological dysregulation, dramatically reducing sleep drunkenness and restoring natural awakening.

Matthew is part of a project working with the Addenbrooke’s Patient‑Led Research Hub and CamRARE to propose a multi‑centre crossover trial to evaluate the medication’s effectiveness and to explore clinical biomarkers for treatment.

Don is the Director for Medicines Development at EATRIS and Scientific Lead of the REMEDi4ALL European Platform for Medicines Repurposing. Don previously headed therapeutic development at the National Center for Advancing Translational Sciences at the US National Institutes of Health following a 30-year career in academia, biotech, non-disease research foundations and patient care organisations.

Olga Solomon holds a degree in Chemistry from the University of Thessaloniki and a Master’s degree in Food Science from the University of Gothenburg. Her professional career spans a wide range of expertise across food safety and pharmaceuticals. 

After five years with a leading beverage company in Greece, she joined the European Commission in 2001. Over the past two decades, she has worked extensively in the field of food safety—covering areas such as food additives, enzymes, and food contact materials—and, for the last twelve years, in pharmaceuticals. 

Since May 2017, she has served as Head of Unit SANTE D.1, “Medicines: policy, authorisation, and monitoring.” In this capacity, she has developed deep knowledge of health policies, risk assessment, risk management, and communication. Throughout her career, she has navigated complex stakeholder landscapes and negotiated proposals with the European Parliament and the Council in both the food and pharmaceutical sectors. 

She has fostered strong partnerships with Member States and European Agencies, including the European Food Safety Authority and the European Medicines Agency. Her influence also extends internationally: she has negotiated food standards within the Codex Alimentarius and led EU activities related to bilateral and multilateral relations in the pharmaceutical field. 

Solomon has guided her team through major preparedness and crisis management efforts, notably during Brexit and the COVID-19 pandemic, including the accelerated authorisation of vaccines and therapeutics. A significant achievement in her career is her central role in shaping the Pharmaceutical Strategy for Europe and leading its implementation, culminating in the adoption of the comprehensive legislative revision package in April 2023. 

Patricia Vandamme is Policy Officer at the Anticancer Fund (ACF). She has worked over 20 years in the pharmaceutical industry, both in local (BE) and global roles, before joining ACF in 2022. As a pharmacist she is fully committed to the health and wellbeing of patients. She plays an important role in connecting with peer organizations, clinical investigators, drug developers and policymakers to make sure drug repurposing makes a leap forward in oncology, as cancer patients urgently need more costly-effective treatments options.

Lydie Meheus is Managing Director of the Anticancer Fund, a Belgian Research Foundation of Public Utility with an international scope. She comes with expertise in clinical research with focus on therapy avenues with limited commercial value but potential societal value. Lydie is also active in European policy work and is well informed about patients’ needs through the free personalized information service offered by the Anticancer Fund and her personal journey.

Lydie co-founded the Anticancer Fund in 2013 with entrepreneur and major funder Luc Verelst, after leading an alike Swiss organisation, Reliable Cancer Therapies, also initiated in 2009 by Luc Verelst.

She obtained a PhD in Sciences in 1986 and worked as head of different research groups and as VP R&D at Belgian biopharmaceutical companies.

Adrian van den Hoven has been director general of Medicines for Europe since September 2013. In his role he focuses on stimulating competition in off-patent medicine markets, fostering access to medicine, reducing medicine shortages and addressing major health crises, supporting policy measures for sustainable pricing, promoting efficient regulatory standards, and developing a coherent EU industrial strategy to support the long-term viability of the generic, biosimilar and value-added medicines industries. He is also member of the European Medicines Verification Organization (EMVO) board for the implementation of serialization against falsified medicines, the International Generic and Biosimilar medicines Association (IGBA) and the joint industry advisory council of the Health Emergency and Response Authority (HERA).

Hugh set up the EHE Rare Cancer Charity UK (EHERCC) in 2015 as his wife has Epithelioid Haemangioendothelioma (EHE), an ultra-rare sarcoma. The charity has raised over £1.25m since inception and has/is funding EHE research in Europe, the US, Australia and the UK, together with EHE-focused foundations in the US, Australia, Italy, and Canada. The charity has also funded the setting up the UK EHE National Biobank, and is helping to fund both a major EHE observational study and an EHE prospective registry across Europe, both managed out of Istituto Nazionale dei Tumori in Milan, Italy. The EHERCC is also part of the working group seeking approval for a label extension for the drug sirolimus for the treatment of EHE. Hugh is married with three children and a labradoodle called Lincoln.

Don is the Director for Medicines Development at EATRIS and Scientific Lead of the REMEDi4ALL European Platform for Medicines Repurposing. Don previously headed therapeutic development at the National Center for Advancing Translational Sciences at the US National Institutes of Health following a 30-year career in academia, biotech, non-disease research foundations and patient care organisations.

Dr Rick Thompson joined Beacon (formerly known as Findacure) as the organisation’s third team member and its first-ever Scientific Officer, with a focus on developing the charity’s work in drug repurposing.

He became CEO in 2017 and has since played a key role in guiding Beacon’s strategy and supporting the delivery of its projects across the rare disease space.

Rick holds a PhD in evolutionary biology and previously studied moles at the University of Cambridge’s Museum of Zoology. He regularly contributes to the European rare disease community through talks, training, and written work, and is continually inspired by the dedication and insight of the patient group leaders that Beacon supports.