Precision Medicine for Rare Diseases” – what seemed like a vision of the future only a few years ago is increasingly becoming reality. We cordially invite you to take a closer look at this forward-looking topic at the 9th Rare Disease Symposium of the Eva Luise and Horst Köhler Foundation.
Precision medicine holds enormous potential for people with rare diseases: faster diagnosis and personalised treatment can significantly improve the quality of life and the course of the disease. At the same time, this progress raises questions: How can patient data be used safely and ethically with small case numbers? How do we organise interdisciplinary collaboration to bring precision medicine to patients with limited evidence and infrastructure? What financing models can support high costs and ensure global access?
These and other questions will be discussed at the symposium. International experts will provide impetus and present current research approaches, technologies and practical solutions. The focus will always be on those affected: Their perspectives and experiences are essential in shaping a sustainable healthcare future. Let’s shape this future together. Share your ideas so that we can make the most of the opportunities offered by precision medicine for rare diseases.
Take a look at the programme here. Registration will start soon!
FRIDAY, JUNE 20
| Time | Topic | Speaker |
| from 8:30 a.m. | Registration and Coffee | |
| 9:00 a.m. | Welcome Address | Eva Luise Köhler |
| 9:05 a.m. | Opening Keynote „Unlocking the Secrets of Rare Diseases: How Precision Medicine is Turning Discovery into Hope” | Professor Anna Greka, Harvard Medical School, Brigham and Women’s Hospital |
| Session 1: Case Study Neuronale Ceroid-Lipofuszinose (NCL) – Understanding and Addressing a Rare Neurological Disorder | ||
| 9:45 a.m. | NCL research: Progress and Challenges | Dr. Angela Schulz, UKE Hamburg |
| 10:05 a.m. | Research Funding for NCL: Strategies and Successes | Dr. Frank Stehr, NCL-Foundation |
| 10:25 a.m. | A Family Perspective on living with NCL | Christian Thulfaut, NCL-Group Germany e.V. |
| 10:45 a.m. | Questions and Discussion | |
| 10:55 a.m. | Coffee Break | |
| Session 2: Unlocking the Power of Data in Rare Diseases | ||
| 11:30 a.m. | Data on Rare Diseases: How to balance the Necessity of Sharing and Privacy? | Prof. Dr. Louisa Specht-Riemenschneider, Federal Commissioner for Data Protection and Freedom of Information |
| 12:00 p.m. | The European Joint Programme on Rare Diseases: Building the Rare Diseases Research Ecosystem | Dr. Daria Julokowska, European Joint Programme on Rare Diseases |
| 12.20 p.m. | UNRARE ME – Establishing New Joint Paths for Patients and Doctors | Prof. Dr. Lorenz Grigull, University Hospital Bonn (UKB) |
| 12.40 p.m. | SATURN — Smart Doctor Portal for Patients with Unclear Diseases | Dr. Jonas Marcello, Fraunhofer Institute for Experimental Software Engineering IESE |
| 1.00 p.m. | Questions and Discussion | |
| 1.10 p.m. | Lunch Break | |
| Session 3: Spotlight Talks: Cutting-Edge Developments – Recognizing, Understanding and Treating Rare Diseases | ||
| 2:15 p.m. | Introduction | Prof. Dr. Annette Grüters-Kieslich, Eva Luise and Horst Köhler Foundation |
| 2.25 p.m. | Next-Generation Genomics – A Cornerstone in Rare Disease Diagnostics | Prof. Dr. Malte Spielmann, University Hospital Schleswig-Holstein (UKSH) (tbc) |
| 2.40 p.m. | Molecular Pathways: Unveiling the Mechanisms of Rare Diseases | Prof. Mehul Dattani, Great Ormond Street Hospital for Children (GOSH) London |
| 2.55 p.m. | Redefining Treatment: Innovations in Gene and Cell Therapies | Prof. Dr. Hildegard Büning, Hannover Medical School |
| 3.10 p.m. | Computationel Systems Biology: Novel Ways for Drug Repurposing | Prof. Dr. Jan Baumbach, UKE Hamburg |
| 3.25 p.m. | Summary, Questions and Discussion | |
| 3.40 p.m. | Coffee Break | |
| Session 4: Looking ahead: Visions and Advances for Tomorrow’s Healthcare of Rare Diseases | ||
| 4:10 p.m. | Precision Medicine: Opportunity or Risk for Vulnerable Groups | Prof. Dr. Claudia Bozzaro, University of Münster |
| 4:40 p.m. | Closing Discussion | |
| 4:55 p.m. | Farewell | Prof. Dr. Annette Grüters-Kieslich, Eva Luise and Horst Köhler Foundation |
| —————————————————– | ||
| 5:00 p.m. | Bus Transfer to the Berlin Brandenburg Academy of Sciences and Humanities | |
| 6:00 p.m. | Ceremonial Presentation of the 17th Eva Luise Köhler Research Award | |
