Scientific Program
| 14 NOVEMBER 2025 | |
| 16:30 - 17:00 | Opening Ceremony and Welcome Address Uğur ÖZBEK, Rumen STEFANOV, Bayram YILMAZ, Rector |
| 17:00 - 18:30 | • Keynote lecture by ICORD representatives, Turkish health officials, and global rare disease advocates. Chair: Uğur Özbek, Rumen Stefanov Speakers: Dave PEARCE - IRDIRC Ahmet AYAR - TİTCK_State Medicine and Medical Devices Agency |
| 15 NOVEMBER 2025 Day 1: Innovations in Rare Diseases |
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| 08:30 - 10:00 | Session 1: Advances in Genomic Medicine Chair: Neşe ATABEY, Munis DÜNDAR, Mehmet ÖZTÜRK |
| 08:30 - 09:00 | • Genomic Diagnostics: Latest technologies for diagnosing rare and undiagnosed diseases. Speaker: Mazhar ADLI, Prof Dr - Northwestern University |
| 09:00 - 09:30 | • International Collaborations: Genome studies from the Middle East. Speaker: Prof. Khalid FAKHRO - Sidra Medicine, Qatar |
| 09:30 - 10:00 | • International Collaborations: Genome studies from Europe: 1MG and GoE Speaker: André UITTERLINDEN - Erasmus MC, NL |
| 10:00 - 10:30 | Coffee Break |
| 10:30 - 12:00 | Session 2: Gene Therapy and Innovative Treatments Chair: Ahmet AYAR, Ferda ÖZKINAY, Salih ŞANLIOĞLU |
| 10:30 - 11:00 | • Platform Therapies: Progress in vector-based gene therapies and their scalability. Speaker: Afrooz RASHNONEJAD - Ohio State Univ (online) |
| 11:00 - 11:30 | • Personalized Medicine: Tailored therapies for ultra-rare diseases (n=1 cases). Speaker: Matt MIGHT |
| 11:30 - 12:00 | • Challenges in Delivery and Access: Logistical and regulatory hurdles in the region. Speaker: Esin Bilge ÖZTÜRK - MeiraGTx, UK |
| 12:00 - 12:30 | SATELLITE SYMPOSIUM |
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| 12:30 - 13:30 | Lunch Break |
| 13:30 - 15:00 | Session 3: Newborn Screening and Early Intervention Chair: Esin KOÇ, Neslihan MUNGAN, Nurten AKARSU |
| 13:30 - 14:00 | • Expanding Newborn Screening: Regional experiences and lessons learned. Speaker: Dr. Peter SCHIELEN chielen - International Society for Neonatal Screening (ISNS) |
| 14:00 - 14:30 | • Preventive Strategies: The role of early diagnosis in reducing disease burden. Speaker: Ahmad N. Abou TAYOUN - Al Jalila Children's Specialty Hospital, UAE |
| 14:30 - 15:00 | • One Health Approach: Integrating human, animal, and environmental health for rare disease prevention. Speaker: Alberto MANTOVANI - Italy |
| 15:00 - 15:30 | Coffee Break |
| 15:30 - 17:00 | Session 4: Patient-Centered Care and Advocacy Chair: Stephen C. GROFT, Pharm. D. - ICORD |
| 15:30 - 16:00 | • Patient Journeys in Rare Diseases: Stories from patients and families, focusing on challenges and resilience. Speaker: Ayşenur ÖZTÜRK |
| 16:00 - 16:30 | • The Role of Advocacy Groups: Highlighting the work of patient advocacy organizations. Speaker: Wilhelm FOUNDATION - Mikk and Helene Cederoth |
| 16:30 - 17:00 | • Panel Discussion: Bridging gaps between patients, healthcare providers, and policymakers. Debaters: Kathy FLYNN - CEO of National-Tay-Sachs and Allied Diseases Dave PEARCE Güneş UYAR Mehmet Nuri ÖĞÜT |
| 17:00 - 18:00 | Networking and Poster / ORAL Presentations |
| • Showcase of local and international research projects, with a focus on regional initiatives. | |
| 16 November 2025 Day 2: Translational Research and Global Collaboration |
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| 08:30 - 10:00 | Session 5: Scientific Breakthroughs and Emerging Therapies Chair: Hamdi AKAN, Mehmet Birhan YILMAZ |
| 08:30 - 09:00 | • Updates on Clinical Trials: Progress in gene therapy and other innovative treatments Speaker: Cynthia TIFFT |
| 09:00 - 09:30 | • CNS Disorders: Advances in therapies for rare neurological conditions. Speaker: V. Göknur HALİLOĞLU - Hacettepe Univ, Türkiye |
| 09:30 - 10:00 | • Molecular Therapies: Novel approaches for inherited inborn errors of metabolism. Speaker: Berna Seker YILMAZ - Ormand Hospital, UK |
| 10:00 - 10:30 | Coffee Break |
| 10:30 - 12:00 | Session 6: Economic and Public Health Perspectives Chair: Rumen STEFANOV, Haluk ÖZSARI |
| 10:30 - 11:00 | • Cost-Effectiveness of Rare Disease Therapies: Balancing innovation and affordability. Speaker: Georgi ISKROV |
| 11:00 - 11:30 | • Public Health Strategies: Integrating rare diseases into national health systems. Speaker: Domenica TARUSCIO |
| 11:30 - 12:00 | • Regional Initiatives: Examples from Turkey, the Balkans, and the Middle East. Speaker: Rumen STEFANOV |
| 12:00 - 12:30 | SATELLITE SYMPOSIUM |
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| 12:30 - 13:30 | Lunch Break |
| 13:30 - 15:00 | Session 7: Education and Capacity Building Chair: Domenica TARUSCIO, Levent ALTINTAŞ |
| 13:30 - 14:00 | • Training Healthcare Professionals: Enhancing skills for rare disease diagnosis and management. Speaker: Manuel POSADA |
| 14:00 - 14:30 | • Patient and Family Education: Empowering patients through knowledge and resources. Speaker: Steve GROFT |
| 14:30 - 15:00 | • Regional Programs: Transferring expertise to Latin America, South Asia, and other regions. Speaker: Emilio ROLDAN |
| 15:30 - 16:30 | Closing Ceremony and Future Directions |
| • Summary of key takeaways and actionable recommendations. | |
| • Announcement of ICORD 2026 location and themes. | |
| • Networking reception. | |