Date
5–6 March 2026
Location
The Cyprus Institute of Neurology & Genetics Nicosia, Cyprus
Language
English
Date
5–6 March 2026
Location
The Cyprus Institute of Neurology & Genetics Nicosia, Cyprus
Language
English
Advancement of Treatments for Rare Diseases
The Cyprus Institute of Neurology & Genetics and the Deputy Ministry of Research, Innovation and Digital Policy, are organising the conference “Advancement of Treatments for Rare Diseases” on 5-6 March 2026, at the Amphitheater, The Cyprus Institute of Neurology & Genetics Nicosia, Cyprus. Rare Diseases affect approximately 30 million people across the European Union and present unique challenges due to their complexity, rarity, and limited treatment options. Advancing research, improving access to innovative therapies, and strengthening collaboration across Europe are essential to improving outcomes for patients and their families.
The two-day event will highlight the EU’s ambition to make Europe a global leader in life sciences by 2030 and will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
THE CONFERENCE
The Conference will feature the following key themes:
- Strengthening EU Collaboration for Rare Diseases
- Advancing Research, Innovation & Therapeutic Development
- Improving Regulatory Frameworks for Orphan Drugs
- Ensuring Accessibility, Equity & Patient-Centred Care
- Enhancing Data Infrastructure, Security & Digital Innovation
WHO WILL ATTEND
The target audience includes government ministries and public authorities; healthcare providers and clinical experts; researchers and the scientific community; representatives from the pharmaceutical and biotechnology industry; patient organisations and advocacy groups; data, digital health, and technology specialists; health economics, ethics, and legal experts; European funding bodies and research agencies; NGOs; and other high-level multi-stakeholder representatives.
Conference agenda
Day 1
Leonidas Phylactou, CEO & Medical Director, CING
TBC
Olivér Várhelyi, EC Health and Animal Welfare Commissioner
Dr. Nicodemos Damianou, Deputy Minister of Research, Innovation and Digital Policy, Republic of Cyprus
Session 1: Current landscape and unmet needs in rare diseases
Carmen Laplaza Santos, Head of Unit Health Innovations and Ecosystems, DG Research & Innovation
Stella Kyriakides, former EC Health Commissioner
Donata Meroni, European Commission DG Sante
Francois Lamy, Vice President of AFM Telethon
Virginie Hivert, Head of Therapies and Access at EURORDIS
Session 2: A framework for developing rationally-designed therapies for rare diseases
Tim Leest, Chair of EMA Committee for Orphan Medicinal Products
Holm Graessner, Lancet Commissioner on Rare Diseases and ERN‑RND Coordinator
Daria Julkowska, Scientific Coordinator of ERDERA
Speakers:
· Tim Leest, Chair of EMA Committee for Orphan Medicinal Products
· Holm Graessner, Lancet Commissioner on Rare Diseases and ERN-RND coordinator
· Daria Julkowska, Scientific coordinator of ERDERA
· Donata Meroni, European Commission DG Sante
· Francois Lamy, Vice President of AFM Telethon
· Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases
Moderator: Dr Carsten Lederer, Senior Scientist, Head of the Blood Disorders Genetics and Thalassaemia Department,The Cyprus Institute of Neurology & Genetics
Session 3: Drug discovery, development and repurposing in rare diseases
TBC
Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology
Priya Singhal, Biogen
Anton Ussi, Coordinator of REMEDI4All
Matthew H. Porteus, Stanford University School of Medicine, Palo Alto
Speakers:
- Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology
- Priya Singhal, Biogen
- Anton Ussi, Coordinator of REMEDI4All
- Matthew H. Porteus, Stanford University School of Medicine
- Kaja Kantorska. Policy officer, DG Sante
Moderator: Dr Petros Petrou, Senior Scientist, Head of the Biochemical Genetics Department, The Cyprus Institute of Neurology & Genetics
Day 2
Session 4: Becoming trial-ready for rare diseases therapies
Helene Le Borgne, Policy Officer, DG Research and Innovation
Maurizio Scarpa, Coordinator of MetabERN
Andri Papadopoulou, Scientific Officer, Joint Research Centre
Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank
Allan Fred Byamukama
Speakers:
- Helene Le Borgne, Policy Officer, DG Research and Innovation
- Maurizio Scarpa, Coordinator of MetabERN
- Andri Papadopoulou, Scientific Officer, Joint Research Centre
- Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank
- Allan Fred Byamukama
- Virginie Hivert, Head of Therapies and Access at EURORDIS
- Alexis Arzimanoglou, Coordinator of ERN EpiCARE
Moderator: Prof. Kleopas Kleopa, Senior Neurologist, Head of the Neuroscience Department, The Cyprus Institute of Neurology & Genetics
Session 5: Rollout of efficacious therapies: access and infrastructure considerations
Neophytos Charalambides, Minister of Health, Republic of Cyprus
Anne‑Sophie Lapointe, French representative in ERN Board of Member States
Christine Leopold, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center
Panikos Voskos, President MDA Cyprus
Speakers:
· Neophytos Charalambides, Minister of Health, Republic of Cyprus
· Anne-Sophie Lapointe, French representative in ERN Board of Member States
· Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center
· Panikos Voskos, President MDA Cyprus
· Stella Kyriakides, former EC Health Commissioner
Moderator: Dr Menelaos Pipis, Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics
Leonidas Phylactou, CEO & Medical Director, CING