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 the people living with a rare disease: 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.
FRIDAY, JUNE 20
KARL STORZ SE & Co. KG Besucher- und Schulungszentrum Berlin (Scharnhorststraße 3, 10115 Berlin)
| Time | Topic | Speaker |
|---|---|---|
| 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” | Prof. Anna Greka, Harvard Medical School |
| Session 1: Case Study NCL | ||
| 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: Balancing the Necessity of Sharing and Privacy | Dr. Sahra Golghalyani |
| 12:00 p.m. | The European Joint Programme on Rare Diseases | Dr. Daria Julkowska |
| 12:20 p.m. | UNRARE ME – Joint Paths for Patients and Doctors | Prof. Dr. Lorenz Grigull |
| 12:40 p.m. | SATURN — Smart Doctor Portal for Unclear Diseases | Dr. Andreas Jedlitschka |
| 1:00 p.m. | Questions and Discussion | |
| 1:10 p.m. | Lunch Break | |
| Session 3: Spotlight Talks on Rare Diseases | ||
| 2:15 p.m. | Introduction | Prof. Dr. Annette Grüters-Kieslich |
| 2:25 p.m. | Next-Gen Genomics in Rare Disease Diagnostics | Prof. Dr. Malte Spielmann |
| 2:40 p.m. | Molecular Pathways: Mechanisms of Rare Diseases | Prof. Mehul Dattani |
| 2:55 p.m. | Innovations in Gene and Cell Therapies | Prof. Dr. Hildegard Büning |
| 3:10 p.m. | Computational Systems Biology and Drug Repurposing | Prof. Dr. Jan Baumbach |
| 3:25 p.m. | Summary, Questions, and Discussion | |
| 3:40 p.m. | Coffee Break | |
| Session 4: Visions for Future Rare Disease Healthcare | ||
| 4:10 p.m. | Precision Medicine: Opportunity or Risk? | Prof. Dr. Claudia Bozzaro |
| 4:40 p.m. | Closing Discussion | |
| 4:55 p.m. | Farewell | Prof. Dr. Annette Grüters-Kieslich |
| 5:00 p.m. | Bus Transfer to Berlin Brandenburg Academy of Sciences and Humanities | |
| 6:00 p.m. | Ceremonial Presentation of the 17th Eva Luise Köhler Research Award | |
