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.
Preliminary Programme
Day 1
Leonidas Phylactou, CEO & Medical Director, CING
Prof. Michael Komodromos, President of the Board of Directors of The Cyprus Institute of Neurology & Genetics
Olivér Várhelyi, EC Health and Animal Welfare Commissioner (pre-recorded video)
Dr. Nicodemos Damianou, Deputy Minister of Research, Innovation and Digital Policy, Republic of Cyprus
Mr. Neophytos Charalambides, Minister of Health of the 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
Dr. Milena Angelova, Member at the European Economic and Social Committee; rapporteur of the opinion
Tim Leest, Chair of EMA Committee for Orphan Medicinal Products
Virginie Hivert, Acting CEO and Head of Therapies and Access at EURORDIS
Speakers:
- Carmen Laplaza Santos, Head of Unit Health Innovations and Ecosystems, DG Research & Innovation
- Stella Kyriakides, former EC Health Commissioner
- Dr. Milena Angelova, Member at the European Economic and Social Committee; rapporteur of the opinion
- Francois Lamy, Vice President of AFM Telethon France
- Virginie Hivert, Acting CEO and Head of Therapies and Access at EURORDIS
Moderator: Dr Menelaos Pipis, Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics
Session 2: A framework for developing rationally-designed therapies for rare diseases
Francois Lamy, Vice President of AFM Telethon France
Donata Meroni, European Commission DG Sante
Dr. Holm Graessner, Lancet Commissioner on Rare Diseases and ERN-RND coordinator
Dr. Daria Julkowska, Scientific coordinator of ERDERA
Dr. Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases
Speakers:
- Tim Leest, Chair of EMA Committee for Orphan Medicinal Products
- Donata Meroni, European Commission DG Sante
- Dr. Holm Graessner, Lancet Commissioner on Rare Diseases and ERN-RND coordinator
- Dr. Daria Julkowska, Scientific coordinator of ERDERA
- Dr. Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases
- Kaja Kantorska, Policy officer, DG SANTE
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
Dr. Priya Singhal, Head of Development, Biogen
Dr. Kleopas Kleopa, Senior Neurologist, Head of the Neurosciences Department, The Cyprus Institute of Neurology & Genetics
Dr. Matthew H. Porteus, Stanford University School of Medicine, Stanford, USA
Dr. Nicolas Nicolaides, University of Cyprus
Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology
Harald Schmidt, Coordinator of REPO4EU
Anton Ussi, Coordinator of REMEDI4All
Prof. Birute Tumiene, Assoc Prof. at Vilnius University, Faculty of Medicine (online)
Speakers:
- Dr. Priya Singhal, Head of Development, Biogen
- Dr. Kleopas Kleopa, Senior Neurologist, Head of the Neurosciences Department, The Cyprus Institute of Neurology & Genetics
- Dr. Matthew H. Porteus, Stanford University School of Medicine, Stanford, USA
- Dr. Nicolas Nicolaides, University of Cyprus
- Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology
- Anton Ussi, Coordinator of REMEDI4All
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
Dr. Andri Papadopoulou, Scientific Officer, Joint Research Centre, European Commission
Mr. Spyros Polyviou, Director of the Cyprus Alliance for Rare Disorders
Dr. Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank
Dr. Luca Sangiorgi Director Rare Bone Disorders Dept. and Coordinator Rare Bone Diseases Centre Istituto Ortopedico Rizzoli, Coordinator of ERN-BOND
Allan Fred Byamukama
Speakers:
- Dr. Helene Le Borgne, Policy Officer, DG Research and Innovation
- Dr. Maurizio Scarpa, Coordinator of MetabERN
- Dr. Andri Papadopoulou, Scientific Officer, Joint Research Centre
- Charalambos Papadopoulos, President of the Cyprus Alliance for Rare Disorders
- Dr. Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank
- Dr. Luca Sangiorgi Director Rare Bone Disorders Dept. and Coordinator Rare Bone Diseases Centre Istituto Ortopedico Rizzoli, Coordinator of ERN-BOND
- Allan Fred Byamukama
- Prof. 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
Dr. Anne‑Sophie Lapointe, French representative in ERN Board of Member States
Prof. Nikolaos Zamboglou, German Medical Institute, Limassol
Dr. Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center
Panikos Voskos, President of Muscular Dystrophy Association, Cyprus
Speakers:
- Dr. Anne-Sophie Lapointe, French representative in ERN Board of Member States
- Prof. Nikolaos Zamboglou, German Medical Institute, Limassol
- Dr. Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center
- Panikos Voskos, President of Muscular Dystrophy Association, Cyprus
- Marina Vasiliou, President and Managing Director, Biogen
Moderator: Dr. Menelaos Pipis, Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics
The role of CING in caring for patients with rare diseases: clinical excellence and research innovation
Prof. Leonidas A. Phylactou, CEO & Medical Director of The Cyprus Institute of Neurology & Genetics
The Cyprus Institute of Neurology & Genetics