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SUMMARY:Undiagnosed Day 2026 | Where diagnosis becomes hope: The power of collaboration and technology in rare diseases
DESCRIPTION:April 29-30\, 2026 | Gdansk\, Poland\nUndiagnosed Day 2026 is a two‑day\, clinician‑led meeting in Gdansk bringing together specialist clinicians\, clinical geneticists and invited experts to support phenotype‑led diagnosis through live case discussion\, shared clinical reasoning and practical exchange. \nThe event is organised by the European Rare Diseases Research Alliance (ERDERA); the Wilhelm Foundation; the Medical University of Gdansk (GUMed); and the University Clinical Centre in Gdansk (UCK). \nAlthough it draws on the same collaborative spirit as international Undiagnosed Hackathons\, where patients who have previously failed to receive a diagnosis through standard genetic testing are re-analysed using more advanced techniques\, the Gdansk event is designed around specialist clinical assessment and multidisciplinary discussion with patients and families present. \nWhat to expect\nThe meeting will focus on practical approaches to phenotyping and diagnosis in undiagnosed conditions\, including how to define next steps when a diagnosis remains uncertain\, and how to strengthen pathways and collaboration around undiagnosed care. \nRegister here. \n  \n\n \nPreliminary Programme\n \nWednesday April 29th\, 2026 – CLOSED EVENT\nThis session is not open to the public. \n  \n \nThursday April 30th\, 2026 – OPEN EVENT\nFormat: In‑person – day one closed round table; day two open conference\nVenue: University Clinical Centre in Gdańsk (UCK)\, Centre of Invasive Medicine (CMI)\, Auditorium im. prof. Zdzisława Kieturakisa (nr 2/DD/04). Accessibility map to the venue here. \nTimes are in CEST. \nRegistration for Day 2 is available here.\n \n \n09:00–09:30 – Opening\n\nProf. Tomasz Stefaniak – Director\, UCK Hospital\, Gdansk\nProf. Michał Markuszewski – Rector\, Medical University of Gdansk\nProf. Jolanta Wierzba – Center for Rare Diseases\, Gdansk\nProf. Maria Mazurkiewicz‑Bełdzińska – Department of Developmental Neurology\nMs. Maja Bartoszewicz‑Moritz – Center for Rare Diseases – Patient Council Representative\, Gdansk\n\n \n09:30–09:45 – Introduction\n\nMs. Helene Cederroth – Wilhelm Foundation\nDr. Daria Julkowska  – ERDERA\n\n \n09:45–11:15 – Session I: Modern diagnostics in undiagnosed diseases\nModern diagnostics in undiagnosed diseases brings together clinical genetics\, international research leadership and health‑system perspectives to show how the diagnostic pathway is evolving – from careful phenotyping and functional genomics to scalable data platforms and cross‑border collaboration. \nSpeakers will highlight the global IRDiRC view on accelerating answers\, a practical diagnostic pathway using Cornelia de Lange syndrome as an example\, the current rare‑disease landscape in Poland\, emerging platforms supporting undiagnosed rare conditions (URC) and diagnostic capability‑building\, and how improved diagnostics translate into real‑world care options for patients and families. \nSession Chairs: Prof. Maurizio Scarpa; Prof. Krzysztof Szczałuba \n\nDr. David Pearce – “From Undiagnosed to Rare”; Chair\, International Rare Disease Research Consortium (IRDiRC)\nProf. Frank Kaiser – Diagnostic Pathway of Cornelia de Lange Syndrome; Institute of Human Genetics\, Universitätsklinikum Essen (AöR)\, Essen\, Germany\nProf. Monika Gos – Rare disease situation in Poland; Institute of Mother and Child\, Warsaw\, Poland\nProf. Katrin Ounap – Data platforms for URC and diagnostic capabilities; ERN ITHACA / ERDERA; University of Tartu\, Estonia\nProf. Siddharth Banka – Diagnostics and care possibilities; Manchester Rare Conditions Centre\n\n \n11:15–11:45 – Coffee break\n \n11:45–12:45 – Session II: Clinical cases with and without diagnosis\nClinical cases with and without diagnosis is a case‑based session illustrating the real‑life diagnostic journey in rare and undiagnosed conditions – what enables a breakthrough\, what still blocks answers\, and how precision medicine can (and cannot yet) change management. \nFollowing an introduction to precision medicine\, four cases will be discussed – two solved (each reflecting a long diagnostic odyssey) and two unsolved – to highlight practical decision points\, multidisciplinary collaboration and the added value of advanced genomics\, re‑analysis and data sharing. The session is designed to be interactive\, focusing on transferable lessons for clinicians and the patient community: how to shorten time to diagnosis\, avoid missed opportunities and define next steps when a diagnosis remains elusive. \nSession Chairs: Ms. Shirlene Badger; Dr. David Pearce \n\nProf. Saumya Jamuar – Introduction to precision medicine; Senior Consultant in the Genetics Service at KK Women’s and Children’s Hospital\nProf. Hayane Akopyan – Case report; National Academy of Medical Sciences of Ukraine\, Kyiv\, Ukraine\nDr. Aleksandra Bodetko – Case report; Department and Clinic of Pediatrics\, Endocrinology\, Diabetology and Metabolic Diseases\, Medical University of Wroclaw\, Poland\nDr. Agnieszka Madej‑Pilarczyk – Case report; Department of Medical Genetics\, The Children’s Memorial Health Institute\, Warsaw\, Poland\nProf. Andreas Roos – Adjunct Professor (University of Ottawa); Abteilung für Neuropädiatrie\,\nUniversitätsklinikum Essen\, Germany\nMs. Kornelia Polat – Case report; Student Scientific Circle “Rare Diseases”\, Medical University of Gdansk\, Poland\n\n \n12:45–13:45 – Lunch\n \n13:45–15:15 – Session III: What to do when there is no diagnosis\nWhat to do when there is no diagnosis focuses on practical\, ethical and system‑level actions when a diagnosis is delayed or never achieved. The session connects clinical management (how to organise comprehensive\, coordinated care based on needs rather than labels)\, international practice and patient–family partnership (how “science projects” and collaborative pathways can be pursued responsibly). \nIt will also address privacy and data governance while searching for answers\, and the caregiver burden and quality of life – with and without a confirmed diagnosis – so that support\, communication and services can be planned proactively. The overall aim is to equip participants with a “diagnosis‑agnostic” approach: clear next steps for care\, research participation and family support\, even when certainty remains out of reach. \nSession Chairs: Prof. Frank Kaiser; Dr. Tomasz Grybek \n\nProf. Jan Domaradzki – The Diagnostic Odyssey and Beyond: Caregiver Burden and Quality of Life With and Without Diagnosis; Poznan University of Medical Sciences\, Poznan\, Poland\nProf. Jolanta Wierzba / Dr. Karolina Śledzińska – Diagnosed – and what’s next; comprehensive and coordinated care; Center for Rare Diseases\, Gdansk\, Poland\nProf. Gareth Baynam – Di-Agnostic Rare Care – ONLINE; Rare Care Centre\, Perth Children’s Hospital\, Australia\nMs. Shirlene Badger – When is a diagnosis a diagnosis? Co-creating solutions in patient advocacy; Global Patient Advocacy Lead\, Illumina\nMs. Mary Jane Dykeman – Children’s Privacy: Data Sharing as Possibility not Peril; INQ Law | Health AI\, De‑ID & Applied Innovation | EpiSign Chai\n\n \n15:15–15:45 – Coffee break\n \n15:45–17:15 – Session IV: International cooperation and health data management\nInternational cooperation and health data management explores how cross‑border collaboration\, shared registries and EU‑level infrastructures are reshaping the rare and undiagnosed disease landscape – making data findable and usable\, accelerating diagnostics and enabling more consistent care across countries. \nThe session connects perspectives from ERDERA’s undiagnosed community\, EU coordination and long‑term strategy for rare diseases\, lessons learned from flagship initiatives such as SOLVE‑RD and the role of ERNs. Speakers will also highlight advances in paediatric neurology diagnostics in Poland\, showing how international frameworks translate into real clinical capacity and patient benefit – while keeping ethical boundaries\, privacy and trust at the centre. \nSession Chairs: Prof. Monika Gos; Dr. Karolina Śledzińska \n\nProf. Laima Ambrozaityte – [Speech title tbc]; Dept. of Human and Medical Genetics\, Institute of Biomedical Sciences\, Human Genome and Regulome Research Group\, Translational Health Research Institute\, Faculty of Medicine\, Vilnius University – ERDERA Undiagnosed group\nProf. Maurizio Scarpa – Coordinator\, European Reference Network For Hereditary Metabolic Diseases (MetabERN); Director\, Regional Coordinating\, Center for Rare Diseases\, Udine University Hospital\, Udine\, Italy\nDr. Holm Graessner – When technology meets ethics – the limits and future of transforming diagnosis into therapy for rare diseases; Coordinator of ERN‑RND (Tübingen) and the SOLVE‑RD project “Solving Unsolved Rare Diseases”\nProf. Maria Mazurkiewicz‑Bełdzińska – Advances in diagnostics of paediatric neurology in Poland\n\n \n17:15–17:45 – Closing remarks\n \nContact\nFor practical questions\, please write to global2026@undiagnosed-day.org.
URL:https://erdera.org/event/undiagnosed-day-2026/
LOCATION:80-214 Gdańsk\, M. Smoluchowskiego 17 Centrum Medycyny Inwazyjnej (CMI)\, 80-214 Gdańsk\, M. Smoluchowskiego 17 Centrum Medycyny Inwazyjnej (CMI)
CATEGORIES:Clinical Research,Data Hub,ERDERA,Patient Involvement (PPIE)
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END:VEVENT
BEGIN:VEVENT
DTSTART;TZID=UTC:20251216T150000
DTEND;TZID=UTC:20251216T170000
DTSTAMP:20260501T043733
CREATED:20251021T083545Z
LAST-MODIFIED:20251222T145455Z
UID:10000021-1765897200-1765904400@erdera.org
SUMMARY:Information Webinar - ERDERA Joint Transnational Call 2026
DESCRIPTION:This online webinar will introduce the forthcoming 2026 Joint Transnational Call\, “Resolving unsolved cases in rare genetic and non‑genetic diseases”. Participants will hear how the call fits within ERDERA’s Funding workstream and how it will support multinational\, interdisciplinary projects focused on delivering diagnostic clarity for people living with a rare disease. \nIt will outline scope\, eligibility\, timelines and expectations for patient partnership\, and signpost where to find national/regional rules and submission guidance. \nThe session will also cover how ERDERA’s services and networks can strengthen proposals. A short presentation will introduce ERDERA and its hubs\, including how the Clinical Research Network enhances diagnostic pathways and trial readiness by linking expert centres and harmonising procedures\, the Data Services Hub supports secure\, FAIR data sharing and analysis\, and the Expertise Services Hub offers practical guidance on study design\, regulatory pathways and methods. A dedicated segment on patient engagement will explain how meaningful PPIE is embedded throughout the call—from co‑defining priorities to governance and reporting—so that projects respond to patient‑need and drive real‑world impact. \nPreliminary agenda: \n\nPresentation of ERDERA: who we are and how our hubs support research across Europe;\nJTC 2026 introduction: scope\, eligibility\, timelines and evaluation;\nPatient engagement in research: expectations\, good practice and support for PPIE;\nExpertise support services: how to access methodological\, regulatory and clinical guidance to strengthen your consortium and proposal.\n\nPlease note that spots for this event are limited. If the link does not allow you to register\, please keep checking this website for further updates on how to receive the recording of the event.
URL:https://erdera.org/event/information-webinar-erdera-joint-transnational-call-2026/
CATEGORIES:Clinical Research,Data Hub,ERDERA,Expertise Services,Funding
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LOCATION:
END:VEVENT
BEGIN:VEVENT
DTSTART;VALUE=DATE:20251209
DTEND;VALUE=DATE:20251211
DTSTAMP:20260501T043733
CREATED:20251013T095036Z
LAST-MODIFIED:20251222T151705Z
UID:10000018-1765238400-1765411199@erdera.org
SUMMARY:2nd International Conference on Clinical Research Networks: Connected for impact
DESCRIPTION:ERDERA – the European Rare Diseases Research Alliance – is pleased to announce the upcoming 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases\, co-organised in collaboration with Rare Disease International and the International Rare Diseases Research Consortium (IRDiRC). \nDetails: \nDate: 9–10 December 2025 \nFormat: Hybrid – online and in-person participation available \nVenue: Marsilius College\, Heidelberg\, Germany \nView the speaker bios here\n  \nProgramme\nTimes are in CET. \nTuesday December 9th\, 2025\n12.00 – 13.00 – Light lunch reception\n13.00 – 14.00 – Plenary session: Opening Session – Setting the scene\n\nDavid Pearce\, International Rare Diseases Research Consortium (IRDiRC) Professor Department Pediatrics\, Sanford School of Medicine of the University of South Dakota (United States of America)\nAlexandra Heumber Perry\, Chief Executive Officer\, Rare Diseases International (France)\nBaptiste Eluard\, Senior Scientific Project Manager at the European Rare Disease Research Alliance (ERDERA)\n\n14.00 – 15.30 – Plenary session 1: Global efforts in Real World Evidence (RWE) and data collection\nSession Chair: Franz Schaefer\, Hospital Franz Schaefer\, MD\, Professor of Pediatrics Head\, Division of Pediatric Nephrology\, Heidelberg University Coordinator\, European Reference Network for Rare Kidney Disease (ERKNet) Co-Lead\, Clinical Research Network of the European Rare Disease Research Alliance (ERDERA) \n\nCarla Jonker\, Scientific Administrator\, Real World Evidence workstream (RWE); Data Analytics and Methods Task Force (TDA); European Medicines Agency (EMA) (The Netherlands)\nRamona Walls\, Data Collaboration Center Director\, C-Path (United States of America)\nProf. Thomas Klockgether\, German Center for Neurodegenerative Diseases (DZNE) (Germany)\nPat Furlong\, President\, Parent Project Muscular Dystrophy (United States of America)\n\n15.30 – 16.00 – Coffee and networking break\n16.00 – 18.00 – Plenary session 2: New approaches in diagnostics & clinical research\nSession Chair: Yanis Mimouni (Associate Director\, Regulatory Science\, C-Path) \n\nTudor Groza\, Co-Lead AI and Data Science\, Maternal and Child Health Research Institute\, KK Women’s’ and Children’s Hospital Singapore\, Principal Scientist\, Bioinformatics Institute\, A*STAR (Singapore)\nSergi Aguiló Castillo\, RadboudUMC (The Netherlands)\nAlexander Hoischen\, Radboudumc Nijmegen (The Netherlands)\nCécile Ollivier\, Vice President Global Affairs\, Critical Path Institute (The Netherlands)\nDustin O’Dell\, Cofounder & CEO\, SymetryML Inc and Decentra Health (United States of America)\nChris Hendriksz\, A Rare Cause (South Africa)\nYong Chen\, Ph.D.\, FASA\, FACMI\, Professor of Biostatistics\, Director of the Penn Computing\, Inference and Learning lab\, Founding Director of the Center for Health AI and Synthesis of Evidence (CHASE)\, Perelman School of Medicine at the University of Pennsylvania (USA)\n\n18.30 – 20.30 – Networking reception\nWednesday December 10th\, 2025\n9.00 – 11.00 – Parallel workshops\nPARALLEL WORKSHOP 1: Learnings from Low and Middle-Income Countries: Ensuring Representation in Clinical Research Data and Registries \nSession Chairs: Daria Julkowska\, Scientific coordinator of the European Rare Diseases Research Alliance (ERDERA) and Assistant Director of the Thematic Institute for Genetics\, Genomics & Bioinformatics (IT GGB) at INSERM\,\nMonica Drum\, Senior Global Programmes Manager\, Rare Diseases International (RDI) \n\nChris Hendriksz\, A Rare Cause (United Kingdom / South Africa)\nRoberto Giugliani\, Professor of Genetics at the Federal University of Rio Grande do Sul (UFRGS)\, Chief of the Clinical Research Group in Clinical Genetics at Hospital de Clinicas de Porto Alegre (HCPA)\, and Executive Director of Casa dos Raros\, in Porto Alegre (Brazil)\nRobin Sarfati\, CTO at Tekkare (France)\nProf. Mohamed Hassany\, Assistant Minister of Health for Projects and Public Health Initiatives Professional Affiliation: Ministry of Health and Population (Egypt)\n\nPARALLEL WORKSHOP 2: Rules of engagement in multi-stakeholder pre-competitive environments \nSession Chairs: Samantha Parker Patient Engagement Lead Rare Diseases at Italfarmaco\, Vice-Chair IRDiRC;\nHeidrun Hildebrand\, Alliance Manager Pediatric Development; Bayer Pharmaceuticals \n\nRicardo Fernandes\, Chief Medical Officer at conect4children-stichting\nCécile Ollivier\, Vice President Global Affairs\, Critical Path Institute (The Netherlands)\nBegoña Nafria Escalera\, Head of Patient Engagement in Research Area\, Paediatric Cancer Center Barcelona – PCCB\, Institut de Recerca Sant Joan de Déu (Spain)\nVolker Straub\, Harold Macmillan Professor of Medicine; Director\, The John Walton Muscular Dystrophy Research Centre; Deputy Dean\, Translational and Clinical Research Institute\, Faculty of Medical Sciences\nNewcastle University and Newcastle Hospitals NHS Foundation Trust (United Kingdom)\nKristina An Haack\, MD\, Senior Project Head Rare Development\, Inherited NeuroMetabolic Diseases\, Pediatric Network Lead (France)\nVictoria Hedley\, Together4Rare (United Kingdom)\n\n11.00 – 11.30 – Coffee and networking break\n11.30 – 12.30 – Plenary session 3: Reporting back from parallel workshops & discussion\n\nMonica Drum\, Senior Global Programmes Manager\, Rare Diseases International (France)\nDaria Julkowska\, Scientific coordinator of the European Rare Diseases Research Alliance (ERDERA) and Assistant Director of the Thematic Institute for Genetics\, Genomics & Bioinformatics (IT GGB) at INSERM (France)\nHeidrun Hildebrand\, Alliance Manager Pediatric Development; Bayer Pharmaceuticals (Germany)\n\n12.30 – 13.30 – Lunch break\n13.30 – 15.00 – Plenary session 4: Global trials\, local impact: Empowering access and engagement in international clinical research\, role of patients and broad partnerships\nSession Chair: Alexandra Heumber Perry\, Chief Executive Officer\, Rare Diseases International (France) \n\nDr. Sudheendra Rao\, N R\, Scientific Advisor\, Organization for Rare Diseases India\nChristine Mutena\, Co-Founder at Rare Disorders Kenya\nBegoña Nafria Escalera\, Head – Patient Engagement in Research Area\, Pediatric Cancer Center Barcelona\, Sant Joan de Deu SJD\, Spain; Conect4Children Stichting\nDr Roberto Giugliani\, Professor of Genetics at the Federal University of Rio Grande do Sul (UFRGS)\, Chief of the Clinical Research Group in Clinical Genetics at Hospital de Clinicas de Porto Alegre (HCPA)\, and Executive Director of Casa dos Raros\, in Porto Alegre (Brazil)\n\n15.00 – 15.30 – Coffee and networking break\n15.30 – 17.00 – Plenary session 5: Global networks and models of care in high income and LMIC – The Duchenne experience\nSession Chair: David Pearce\, International Rare Diseases Research Consortium (IRDiRC) Professor Department Pediatrics\, Sanford School of Medicine of the University of South Dakota (United States of America) \n\nAnna Thetford\, Registered Nurse; Program Director (Clinical)\, Rare Care Centre\, Perth\, Western (Australia)\nKarolína Podolská\, MD\, Internal physician and coordinator of Center for adults with muscular dystrophy\, General University Hospital in Prague\, Czech Republic\, Accredited Duchenne Centers Program Manager\nRicardo Fernandes\, Chief Medical Officer at conect4children-stichting\nKarolina Śledzińska\, MD\, PhD\, Pediatrician\, Clinical Geneticist\, Department of Pediatrics\, Hematology and Oncology\, Medical University of Gdansk\, Poland – ONLINE\n\n17.00 – 17.30 – Closing of the conference
URL:https://erdera.org/event/international-conference-on-clinical-research-networks/
CATEGORIES:Clinical Research
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END:VEVENT
BEGIN:VEVENT
DTSTART;TZID=Europe/Brussels:20251120T080000
DTEND;TZID=Europe/Brussels:20251121T130000
DTSTAMP:20260501T043733
CREATED:20251023T134321Z
LAST-MODIFIED:20251222T151641Z
UID:10000020-1763625600-1763730000@erdera.org
SUMMARY:Networking of underrepresented countries for the enhancement of rare disease research
DESCRIPTION:ERDERA invites researchers\, clinicians\, patient representatives\, funders and national authorities from underrepresented countries to a two‑morning virtual workshop focused on strengthening collaboration in rare disease research. Click here for more specific information on what underrepresented countries are. \nThe event will present new evidence from an ERDERA survey across more than 30 countries and co‑create practical solutions to improve inclusion\, networking and capacity development. Outcomes will inform ERDERA guidance for national‑level actions. \nWhy this matters\nPeople living with a rare disease in smaller or less‑resourced countries often face the longest waits for diagnosis and the fewest opportunities to join research and trials. By aligning needs\, sharing good practice and designing realistic support measures\, this workshop aims to accelerate earlier diagnosis and more inclusive research across Europe. \nWhat to expect\n\nEvidence briefing: key findings from ERDERA’s year‑one survey of underrepresented countries\, complemented by insights from related European initiatives (e.g. ERA‑LEARN\, Alliance4Life).\nBest‑practice snapshots: short talks from patients\, clinicians\, researchers and funders\, plus ERDERA workstreams (diagnostics\, clinical trials\, education & training\, National Mirror Groups) on practical measures that work.\nFacilitated parallel sessions: multi‑stakeholder\, multinational groups (using Miro) to identify top challenges on day one and co‑develop actionable solutions on day two across five priorities: policy\, collaboration\, funding\, technical assistance and patient engagement.\nNext steps: synthesis to inform ERDERA guidance for national capacity development in underrepresented countries.\n\nWho should join\n\nParticipants from underrepresented countries: patient organisations\, clinicians\, researchers\, universities\, hospitals\, ministries\, and research‑funding bodies.\nERNs\, SMEs/industry\, and European infrastructures interested in equitable participation and capacity building.\n\nPractical details\n\nDates and time: 20–21 November\, 09:00–13:00 CET each day\nFormat: Online (MS Teams)\nCost: Free; places prioritised for stakeholders from underrepresented countries\nAccessibility: Plain‑language facilitation; collaborative whiteboards; recordings available to registered participants\nRegistration: Register for the workshop\n\nProgramme\n1st day\, November 20th\, 2025\n08:30 – 09:00\nLaunching for on-line participants \n09:00 – 09:15\n\nPresentation of workshop aims and explanation of the foreseen activities\nBirutė Tumienė\, Assoc.Prof.\, MD\, PhD\, Vilnius University\, Faculty of Medicine\, Head of the Division of Clinical Genetics and Genomics\, Centre for Medical Genetics\, Vilnius University Hospital Santaros Klinikos (Lithuania)\n \n09:15 – 09:30\n\nOverview of the survey on the needs of underrepresented countries (UC) in ERDERA T24.1\nBirutė Tumienė\, Assoc.Prof.\, MD\, PhD\, Vilnius University\, Faculty of Medicine\, Head of the Division of Clinical Genetics and Genomics\, Centre for Medical Genetics\, Vilnius University Hospital Santaros Klinikos (Lithuania)\n \n09:30 – 09:45\n\nBridging the Innovation Divide in Europe: The Role of Alliance4Life in Fostering Research Excellence in Central and Eastern Europe\nKarolis Ažukaitis\, Assoc. Prof.\, MD\, PhD\, Vice-Dean for Research at Vilnius University\, Pediatric Nephrologist at Vilnius University Hospital Santaros Klinikos (Lithuania)\n \n09:45 – 10:15\nBest practice examples from: Patients\, clinicians/ researchers: \n· EJPRD project from JTC 2020 ENISNIP\nRobert Jech\, Prof. MD\, PhD\, Head of the Center for interventional treatment of movement disorders\, Department of Neurology\, 1st Faculty of Medicine\, Charles University and General University (Czech Republic) \n· EJPRD project from JTC 2021 SeeMyLife and network support scheme event (European Aniridia Conference 2020)\nArvydas Gelžinis\, MD\, PhD\, Head of the Department of Pediatric Eye Diseases\, The Hospital of Lithuanian University of Health Sciences Kauno klinikos; Irma Bylė\, president of Aniridia Lithuania association (Lithuania) \n10:15 – 12:45\nParallel workshops (supported with MIRO): what are the main challenges for the networking of underrepresented countries and their engagement into rare disease research? \n12:45 – 13:00\nReconvening for the first feedback to plenary \n2nd day\, November 21st\, 2025\n08:30 – 09:00\nLaunching for on-line participants \n09:00 – 10:00\n\nBest practice examples from: Funding agencies: presentation of Lithuanian Research Council;\nŽivilė Ruželė\, PhD\, Advisor at the International Cooperation Unit at Research Council of Lithuania\, ERDERA Networking Support Scheme Secretariat (Lithuania)\n \nERDERA Workstreams\, measures for engagement of underrepresented countries: \n· Diagnostic research workstream (WP6 and WP8)\nProf. Katrin Õunap\, University of Tartu\, Institute of Clinical Medicine & Center of Rare Diseases\, Genetic and Personalized Medicine Clinic\, Tartu University Hospital (Estonia) \n· Education and training (WP20)\nBirutė Tumienė\, Assoc.Prof.\, MD\, PhD\, Vilnius University\, Faculty of Medicine\, Head of the Division of Clinical Genetics and Genomics\, Centre for Medical Genetics\, Vilnius University Hospital Santaros Klinikos (Lithuania) \n· National Mirror Groups (WP23)\nMadara Auzenbaha\, Assoc. Prof. Dr. MD\, lead researcher in Riga Stradins University Scientific Laboratory of Molecular Genetics\, Head of Rare Diseases Coordination Center\, Children’s Clinical University Hospital of Riga (Latvia) \n10:00 – 12:45\nParallel workshops (supported with MIRO): what are the main solutions to enhance networking of underrepresented countries and their engagement into rare disease research? \n12:45 – 13:00\nWrap-up and next steps
URL:https://erdera.org/event/networking-of-underrepresented-countries-for-the-enhancement-of-rare-disease-research/
CATEGORIES:International Alignment,Patient Involvement (PPIE),Training & Education
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LOCATION:
END:VEVENT
BEGIN:VEVENT
DTSTART;VALUE=DATE:20251103
DTEND;VALUE=DATE:20251105
DTSTAMP:20260501T043733
CREATED:20251001T145433Z
LAST-MODIFIED:20251009T141651Z
UID:10000013-1762128000-1762300799@erdera.org
SUMMARY:XVII Foresight Training Course | From research to access: how does Europe speed up the availability of medicines for unmet needs?
DESCRIPTION:The course will focus on the current status and advancements of clinical research especially to address unmet medical needs. Innovative methodologies\, regulatory challenges\, and the involvement of adolescents in clinical trials will be discussed. The state of the art of funding strategies and investments will be reviewed\, considering public incentives and public-private funds at EU\, national and regional level. Access-related issues\, such as joint procurement\, national policies\, HTA\, and patient involvement in decision-making and access procedures will also be covered. \nAdditional details and registration here.
URL:https://erdera.org/event/xvii-foresight-training-course-from-research-to-access-how-does-europe-speed-up-the-availability-of-medicines-for-unmet-needs/
CATEGORIES:Clinical Research,Expertise Services,Funding,International Alignment,Patient Involvement (PPIE),Training & Education
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END:VEVENT
BEGIN:VEVENT
DTSTART;TZID=UTC:20251030T090000
DTEND;TZID=UTC:20251030T232000
DTSTAMP:20260501T043733
CREATED:20250808T125359Z
LAST-MODIFIED:20251010T111046Z
UID:10000011-1761814800-1761866400@erdera.org
SUMMARY:ERDERA’s Open Session - 2nd General Assembly Meeting
DESCRIPTION:ERDERA will mark its 1st anniversary with a stimulating online event open to the entire rare disease community. Leading experts in the field of neurology\, neurometabolic diseases and patient advocacy will take the floor next 30 October\, from 09:00 to 11:20 CET\, bringing both scientific and lived experience to the table. The session is free to attend\, but registration is required. Register here. \nAgenda \n09:00 – 09:10 | Welcome speeches\n• Yvo Roos\, UMC Board Member\, Amsterdam\, The Netherlands\n• Daria Julkowska\, ERDERA Coordinator\, INSERM\, Paris\, France  \n09:10 – 10:00 | Keynote speech (remote)\n• Fanny Mochel\, Director\, Reference Centre for Neurometabolic Diseases\,\nLa Pitié-Salpêtrière Hospital – APHP-Sorbonne University\, Paris\, France  \n10:00 – 10:50 | Keynote speech\n• Bojana Mirosavljevic\, Founder\, Zivot-Life Association\, Belgrade\, Serbia  \n10:50 – 11:20 | One year of ERDERA achievements\n• Daria Julkowska\, ERDERA Coordinator\, INSERM\, Paris\, France  \nInspiring speakers  \n\nFanny Mochel – Professor of Genetics at Sorbonne University\, Mochel leads national reference centres for neurometabolic diseases and leukodystrophies in adults and co-leads the MIND team at the Paris Brain Institute. Her research focuses on brain energy deficiencies\, identifying biomarkers through metabolomics and metabolic imaging\, and developing therapeutic approaches targeting the Krebs cycle. Recently\, she has explored the links between physics and metabolism in brain function and disease. Her work in identifying biomarkers and developing novel treatment strategies reflects ERDERA’s commitment to accelerating scientific discovery and improving patient outcomes. \nBojana Mirosavljević – An internationally recognised rare disease advocate\, scientist\, and patient leader\, Bojana founded the “Life” patient association for children with rare diseases and championed Zoya’s Law\, ensuring early diagnosis and improved care for over 4\,000 patients. With a background in chemistry\, reproductive biology\, and embryology\, she has presented at the European Parliament\, United Nations\, and over 150 global conferences. Currently\, she directs Advocacy Strategy at ICON’s Center for Rare Diseases\, embedding patient perspectives in research and clinical trials worldwide. Her tireless efforts to embed the patient voice into clinical research exemplify ERDERA’s dedication to empowering communities and shaping research that meets real patient needs. \nYvo Roos – Professor of Acute Neurology and dean of the medical faculty at the University of Amsterdam\, Roos pioneered acute stroke care through the founding of the Acute Brain Care Unit in 1996. He has co-led landmark trials including MRCLEAN\, MRCLEAN No-IV\, and DIRECT MT\, and leads large research collaborations such as the CONTRAST consortium and CINTICS. With over 450 publications and multiple prestigious awards\, his work has shaped stroke care and research internationally. His work mirrors ERDERA’s vision of fostering multi-centre\, cross-disciplinary collaborations to translate research into meaningful clinical impact. \nDaria Julkowska – Scientific Coordinator of the European Rare Disease Research Alliance (ERDERA) and Assistant Director of the Thematic Institute for Genetics\, Genomics & Bioinformatics at INSERM\, with over 15 years of experience in research and management. Julkowska has developed and implemented collaborations between European research networks\, infrastructures\, and patient organisations. With a PhD in Molecular Biology\, postdoctoral experience in cellular biology\, and an MSc in Management of Research\, she combines scientific expertise with strategic coordination to advance rare disease research across Europe. \n\nRegister now! \nDon’t miss the chance to be part of an inspiring occasion at the ERDERA Open Session on 30 October 2025\, from 09:00 to 11:20 CET.      \nThe session is free to attend\, but registration is required. You can register to attend the open session here.
URL:https://erdera.org/event/erderas-open-session-nd-general-assembly-meeting/
CATEGORIES:ERDERA
ATTACH;FMTTYPE=image/jpeg:https://erdera.org/wp-content/uploads/2025/09/Erdera-Open-Session-1-1.jpg
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