Learn from world KOLs in rare diseases on the latest in diagnosis, AI, NBS, gene therapy, clinical trials and patient access. Ensure that you are equipped with the most recent policy and governmental changes in OMP legislation and EUHTA.
With a footfall of 2000+ attendees, exhibitors will get to speak directly to their target market, expand sales leads, strengthen and establish their brand and close deals with customers on-site.
Get access to our dedicated event app (3 weeks before the event), see all of our 2000+ attendees and set up 1-2-1 meetings to maximise your ROI in attending the congress.
Meet start-ups or pitch your company at the leading rare disease congress. Meet new companies that can help your business or investors that can take you to the next level.
Host or participate in our pre-congress workshops where we deep dive into topics including AI, early access, patient journey, RD action plan and EU joint clinical assessment with all stakeholders.
Be part of a NEW format – our working groups. Replacing roundtables, these sessions are intimate discussions and one of the best ways to find out what your target audience wants.
Our patient zone provides you with the unique opportunity to engage with Patient Advocacy Groups and ERNs who are at the heart of the rare disease community.
The media zone will be bustling with all of the who’s who in the rare disease world. Witness live interviews and use our zone to announce press releases and social media outreach to maximise your presence.
Join 2,000 attendees at the world's largest orphan drug and rare disease event.
This is your best opportunity in 2024 to network with like-minded professionals.
Claire holds a PhD in biochemistry and has led organisations that champion and advocate for commercial delivery of science for 25 years, starting within the Cambridge UK biotechnology cluster. She has worked to champion biotechnology across sectors through the Council of European BioRegions and the ...
Dr Mary Wang is Programme Director at Rare Diseases International (RDI) where she leads RDI’s Global Programme on diagnosis, development and access to essential therapies, and oversees RDI’s technical collaboration with the World Health Organization. Mary has 20 years of experience across sectors...
DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...
Kate Holliday is an advanced practice nurse in rare and genetic conditions, patient experience researcher and community engagement expert. She has worked in the health sector for more than 20 years. In 2020, Kate was listed on the 100+ Outstanding Women Nurse and Midwife Leaders by Women in Global...
Frauke Naumann-Winter is a molecular biologist and epidemiologist by training and holds a PhD in Genetics. Frauke Naumann-Winter is currently Head of Unit for Paediatric and Orphan Medicinal Products in the Federal Institute for Drugs and Medical devices and is the German delegate and Vice-Chair at ...
Francis Pang is Senior Vice President, Global Market Access and International Geographic Expansion at Orchard Therapeutics and has more than 20 years of experience in pricing and reimbursement, market access, health economics and corporate development, encompassing leadership roles at Shire Human...
Rachel has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development ...
Lara Bloom is the President and CEO of The Ehlers-Danlos Society and responsible for globally raising awareness of rare, chronic, and invisible diseases, specializing in the Ehlers-Danlos syndromes (EDS), hypermobility spectrum disorders (HSD), and related conditions. Lara leads coordinated medical ...
Dr Kirsten Johnson is the President and one of the founders of Fragile X International. She also sits on the boards of EURORDIS - Rare Diseases Europe.Kirsten is a FMR1 premutation carrier and has two adult daughters who live with Fragile X Syndrome. Her husband lives with a rare cancer, hairy cell ...
Researcher at the Health Technology Assessment Area (AETSA) of the Regional Ministry of Health and families of Andalusia, Seville, Spain. Degree in Physiotherapy and a master´s degree in Motor Neurocontrol. Anabel is currently working a PhD in Clinical Procedures, Interventions, and Health Outcomes ...
Doctor in Medicine. Paediatric Neurologist in the Hospital Sant Joan de Déu in Barcelona, Spain. Specialist in movement disorders. He is member of the Spanish Society of Pediatric Neurology, the European Pediatric Neurology Society, the Society for the Study of Inborn Errors of Metabolism and the...
With over 10 years of experience in the pharmaceutical industry, Ana is a recognized expert in the field of Expanded Access Programs (EAP). Her expertise lies in oncology, hematology, Rare Diseases, and she has held various roles in the big Pharma (Roche, Menarini-Stemline), including leading asset ...
Sarka Kubinova, PhD, is the coordinator of the MoCA (Mechanism of Coordinated Access to Orphan Medicinal Products) project. She currently works as an HTA assessor at the State Institute for Drug Control, the national regulatory authority for pharmaceuticals in the Czech Republic. Prior to this, she ...
Thomas Bols | Head of Government Affairs and Patient Advocacy, EMEA, PTC Therapeutics and board member of Europabio Thomas has been a board member of Europabio since June 2022 and chairs a variety of industry federation working groups. In the past he held other board positions at several European ...
Degree in Pharmacy. Postgraduate degree in public health. Postgraduate in Management and Strategic Planning for Non-Profit Organisations. President of ASMD SpainBoard member of FEDER (Spanish Federation of Rare Diseases).Board member of Eurordis (Rare Diseases Europe).After working for more than 20 ...
Julien Delaye joined EURORDIS in September 2019 as an intern and moved to the position of Public Affairs Assistant on the Rare 2030 project. He then became Public Health Policy Junior Manager and now holds the position of Patient Engagement Manager in HTA. Julien is responsible for organizing the...
Laura I. Furlong is Co-founder and Scientific Director of MedBioinformatics. She holds a PhD in Biology from the University of Buenos Aires and an MSc in Bioinformatics from Pompeu Fabra University. With more than 18 years of experience in translational bioinformatics, one of her main interests is...
Hasan is a pharmacist with a profound expertise in clinical oncology. Armed with a PhD in this specialized field, he has devoted years to conducting real-world data studies that delve into the effectiveness and safety of cancer drugs in practical, everyday healthcare scenarios. He has experience in ...
Jonathan Kornstein is Vice President, Rare Disease and Pediatrics at Caidya and has over 29 years of expereince in the Pharmaceutical and CRO industry. He has demonstrated a depth of project management and clinical operations experience in a multitude of therapeutic areas with a focus on rare and...
Cristina Montané, linked to patient associations in which she has worked as a volunteer for more than twenty years. She completed the Postgraduate Course in Patient Advocacy at the UIC and the UCM. She is a board member of the Catalan Patient Advisory Council, Citizen Participation Director of the...
Emilie has six years of experience in rare disease market access. Areas of expertise include strategy development, policy (including revision of the General Pharmaceutical Legislation), access in low- and middle-income countries and value communication. Prior to joining Dolon, Emilie obtained a BSc ...
Michele Pistollato, PhD is a Principal at Charles River Associates. He specialises on policy topics in Life Sciences, ranging from value assessment and pricing dynamics for pharmaceuticals to public policy issues regarding specific therapy areas. For over 10 years he has worked extensively on the...
Having been with Clinigen for over 12 years now, I am currently responsible for providing Regulatory advice both internally at Clinigen Group, but also working with our clients to compliantly manage their regulatory strategy for managed / early access programmes. Liaising with experts and Competent ...
Alison Cave is Chief Safety Officer at the Medicines and Healthcare products Regulatory Agency. She holds a BSc Honours degree and PhD and has significant academic research and regulatory experience with roles at Kings College London, the Wellcome Trust, UK Research and Innovation and the European...
Nitin Mahadev has over 25 years of experience and expertise in pharmaceuticals. He currently serves as the Global Gene Therapy and Hemophilia Lead at Pfizer and is responsible for managing a portfolio of in-line hemophilia assets and leading the launch and commercialization of Pfizer’s pipeline in...
Monica has worked across the community, health, education, business and government sectors. Monica is a Council Member of Rare Disease International and currently serving the genetic, undiagnosed and rare disease communities as the Chief Executive, Genetic Support Network of Victoria (GSNV). She is ...
General Director, Directorate-General for the Common Catalogue of Services of the National Health System and Pharmacy, Spanish Ministry of Health . From 2009-2022, Head of Medicines for Human Use Department- Spanish Medicines Agency . PhD in Medicine (Complutense University of Madrid) specialised...
A dedicated advocate for rare disease patients, Toni co-founded DEBRA South Africa after years of connecting and supporting individuals affected by Epidermolysis Bullosa (EB) around the country. Armed with her degrees in Psychology and 10-year experience as a qualified Life Line lay counsellor, she ...
Dr. Arantxa Sancho is the Director of the Medical-Scientific Affairs Department at Farmaindustria. She holds a degree in Medicine and Surgery from the Autonomous University of Madrid and a PhD in Pharmacology. Her extensive experience encompasses both research and regulatory affairs. Before joining ...
Robin Marcus is Marken’s Head of Global Decentralized Trials Market Development and has over 30 years of experience driving business optimization, championing innovation and generating aggressive revenue growth. She has successfully launched new products, divisions and companies that have exceeded...
Dr. Ina Sarel is a biotechnology executive with over 20 years of experience in product development from discovery and Proof of Concept through pre-clinical and clinical stages. Ina has broad expertise in stem/progenitor cell therapy, CMC, and regulatory requirements and previously developed a stem...
After a Phd in Philosophy and a first experience in a competitiveness-focus Brussels think tank, Matteo Scarabelli joined Eurordis - the European Organisation of rare disease patients - to be responsible for the involvement of patients in research and decision making for orphan drugs. For two years ...
PhD qualified with 20+ years of experience in Basic Research, Clinical Trial Monitoring, Patient Registry design and monitoring, Healthcare Policies design and implementation, Multidisciplinary Reference Units accreditation and monitoring, Patient Advocacy and Bioethics, within an academic Health...
Dr. Levine, President Fondation Ipsen, has three decades of experiencein the healthcare sector principally at Mayo Clinic. For the last 6 years hehas been President of Fondation Ipsen, an international science foundationfocused on biotech innovation in Rare Diseases.A physician and scientist, James ...
Mila is a Senior Manager in Clinical Operations at Allucent, with over 15 years of experience in clinical research and operations. Throughout her career, she has worked in Immunology, Microbiology, and Molecular Biology, providing her with extensive knowledge in the development of clinical trials...
Wendy Erler is the Vice President of Patient Experience, Patient Advocacy and Patient Insights at Astra Zeneca, Alexion Rare Disease where she is responsible for leading the enterprise innovation model to gather and incorporate patient insights throughout the drug development and commercialization...
Ross’s drive is to enable patient access to medicine – a prime professional responsibility as a clinical pharmacist. An early career in the NHS specialising in renal, haematology and neonatal medicine enabled him to understand complex medicine and therapeutics and bridge individualised patient care ...
Doctor in Medicine and Health Economist, he worked in various positions in the public sector (CatSalut) before founding the first European company in Health Economics and Outcomes Research, which was later acquired by IMS Health. He then joined IMS’s Senior Leadership Team in Europe. With over 35...
Maria Judit Molnar, MD, PhD, is a professor of neurology, psychiatry, clinical genetics, and clinical pharmacology, and a doctor of the Hungarian Academy of Sciences. She serves as the director of the Institute of Genomic Medicine and Rare Disorders at Semmelweis University and is a member of the...
David Pearce is the chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC) that represents funding agencies, companies, and patient advocate groups that from approximately 80 countries. He completed his undergraduate Bachelor of Science Degree with honors ...