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

Dear delegates,

In light of the latest developments and as a precautionary measure, the Cyprus Presidency of the Council of the EU 2026 has decided to postpone this week’s Presidency Conference Advancement of Treatments for Rare Diseases, scheduled to take place in Cyprus 5–6 March 2026.

Further information regarding possible re-arrangement and new dates will be communicated in due course.

We apologise for the inconvenience caused by these adjustments and thank you for your understanding.

Yours sincerely,
The Organizing Committee
Conference “Advancement of Treatments for Rare Diseases”
Cyprus Presidency of the Council of the European Union 2026

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.

Speakers

Eftyhia Andreou

Teacher , Journalist , Reporter , Presenter

Leonidas Phylactou

Chief Executive Officer & Medical Director, The Cyprus Institute of Neurology & Genetics

Michael Komodromos

President, The Cyprus Institute of Neurology & Genetics

Olivér Várhelyi

Commissioner, EC Health and Animal Welfare, European Commission

Nicodemos Damianou

Deputy Minister of Research, Innovation and Digital Policy, Republic of Cyprus

Neophytos Charalambides

Minister of Health, Republic of Cyprus

Stella Kyriakides

Former Commissioner for Health and Food Safety, European Commission

Milena Angelova

Member, European Economic and Social Committee, Raporteur of the opinion

Francois Lamy

Vice president, AFM-Téléthon

Virginie Hivert

Acting Chief Executive Officer and Head of Therapies & Access, EURORDIS - Rare Diseases Europe

Valentina Bottarelli

Head of Policy and Public Affairs, EURORDIS - Rare Diseases Europe

Menelaos Pipis

Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics

Tim Leest

Chair, EMA Committee for Orphan Medicinal Products

Donata Meroni

Head of Unit, ‘Health Monitoring and Cooperation, Health Networks, European Commission

Holm Graessner

Managing Director, Centre for Rare Diseases, University Hospital Tübingen, Germany and Coordinator of ERN-RND

Daria Julkowska

Scientific coordinator, European Rare Diseases Research Alliance (ERDERA) & Assistant Director, Thematic Institute for Genetics, Genomics & Bioinformatics (IT GGB), INSERM

Violetta Anastasiadou

Chair, Cyprus National Committee for Rare Diseases

Kaja Kantorska

Biotechnologist, DG SANTE, European Commission,

Carsten W. Lederer

Senior Scientist, Head, Blood Disorders Genetics and Thalassaemia Department,The Cyprus Institute of Neurology & Genetics

Priya Singhal

Executive Vice President and Head of Development, Biogen

Kleopas A. Kleopa

Senior Neurologist, Head of Neuroscience Department, The Cyprus Institute of Neurology & Genetics

Matthew Porteus

Professor, Stanford University

Nicolas Nicolaides

University of Cyprus

Demetris Skourides

Chief Scientist for Research, Innovation and Technology, Republic of Cyprus

Anton Ussi

Coordinator of REMEDI4All

Birute Tumiene

Vilnius University, Faculty of Medicine / Hospital Santatos Klinikos, Coordinator for Competence Centres and Member of MetabERN

Petros Petrou

Senior Scientist, Head of the Biochemical Genetics Department, The Cyprus Institute of Neurology & Genetics

Hélène Le Borgne

Policy Officer, DG Research and Innovation, European Commission

Maurizio Scarpa

Paediatrician and Director, Regional Coordinating Centre for Rare Diseases, University Hospital of Udine, Italy Coordinator of MetabERN

Andri Papadopoulou

Scientific Officer, Joint Research Centre, European Commission

Giedrė Kvedaravičienė

Director of the Lithuanian Population and Rare Diseases Biobank

Luca Sangiorgi

Director Rare Bone Disorders Dept. and Coordinator Rare Bone Diseases Centre Istituto Ortopedico Rizzoli, Coordinator of ERN-BOND

Allan Byamukama

Executive Director, Founder, Byamukama Sickle Cell Foundation

Alexis Arzimanoglou

ERN EpiCARE Coordinator and Director of Paediatric Epilepsy Dpt.University Children’s Hospital San Juan de Dios Barcelona, Spain

Nikolaos Zamboglou

Chief Executive Officer, German Oncology Center and the German Medical Institute

Anne‑Sophie Lapointe

French representative in ERN Board of Member States

Christine Leopold

Assistant Professor, Drug Regulatory Science, Division of Pharmacoepidemiology & Clinical Pharmacology, Utrecht Institute for Pharmaceutical Science / WHO Collaborating Centre for Pharmaceutical Policy and Regulation

Panikos Voskos

President of Muscular Dystrophy Association, Cyprus

Marina Vasiliou

President and Managing Director, Biogen France

Spyros Polyviou

Director, Alliance for Rare Disorders, Republic of Cyprus

Harald Schmidt

Coordinator of REPO4EU

Programme

Day 1

08:00- 08:30
Registration and coffee
08:35- 08:40
Welcome Address and endorsement

(pre-recorded video)

 
 
08:50-09:00
Overview of the Rare Disease Landscape in Cyprus

Session 1: Current landscape and unmet needs in rare diseases

09:05-09:20
European research on rare diseases: a long-standing commitment to make Europe a leader at global level
09:20-09:35
Witnessing and implementing change over the past 2 decades: the European Commission’s action on rare diseases and therapy development
09:35-09:45
The European Economic and Social Committee’s exploratory opinion on the advancement of treatments for Rare Diseases
09:45-10:00
Regulatory considerations for rare disease drug development and approval
10:00- 10:15
EURORDIS’ role in facilitating therapies for rare diseases (online)
10:45- 11:15
Coffee Break

Session 2: A framework for developing rationally-designed therapies for rare diseases

11:15- 11:30
Funding and patient advocacy organisations spearheading the effort against rare diseases
11:30- 11:45
A European inventory of rare diseases through the scope of ERNs
11:45- 12:00
The current state of affairs for developing rational therapies for rare diseases
12:15 – 12:25
European Networking for Rare: Equity or Justice?
13:15-14:15
Lunch break

Session 3: Drug discovery, development and repurposing in rare diseases

14:15- 14:30
Building on knowledge and lessons learnt: developing Nusinersen for SMA (online)
14:30 – 14:45
Developing virally-delivered genetic therapies for inherited neuromuscular disorders
14:45- 15:00
A Journey to Developing Curative Therapies for Hemoglobinopathies using Genome Editing
15:00 – 15:15
Developing therapies for rare paediatric endocrine disorders
15:15-15:30
Coffee Break
15:30-15:45
Chasing Miracles of Science: Accelerating Innovation in Rare Disease Medicines
15:45-16:00
The role of artificial intelligence in drug discovery and repurposing
16:00-16:10
The End of Medicine As We Know It
16:10-16:20
Innovative platform-based approach for sustainable repurposing of medicines for rare diseases
16:20-16:30
A clinical model for interdisciplinary sharing of knowledge and innovation for the development of rare diseases therapies (online)

Day 2

08:00- 08:30
Registration and coffee

Session 4: Becoming trial-ready for rare diseases therapies

08:30- 08:45
Introducing initiatives to bring more clinical trials to Europe
09:00- 09:15
The European Platform on rare disease registration: Connecting data, accelerating care pathways and enabling research
09:15 – 09:30
Amplifying patient and public involvement in small rare diseases ecosystems
09:30- 09:45
National Biobanks for Population and Rare Disease Research
09:45 – 10:00
Considerations in the development of sensitive outcome measures needed to track the natural history of rare diseases (online)
10:00- 10:15
Clinical trials from the patients’ perspective
11:00-11:30
Coffee Break

Session 5: Rollout of efficacious therapies: access and infrastructure considerations

11:30- 11:45
Infrastructure considerations for delivering therapies at scale: France’s example
11:45 – 12:00
Capacity building and interdisciplinary training for delivering novel therapies in secondary care comprehensive healthcare facilities
12:15- 12:30
Rare Diseases,Research, Therapies and Social Impact
13:15-13:30
Closing Lecture

The role of The Cyprus Institute of Neurology & Genetics in caring for patients with rare diseases: clinical excellence and research innovation

 

13:30 – 14:30
Lunch Break
14:30–15:30
Tour at the premises of The Cyprus Institute of Neurology & Genetics
15:30–17:00
Farewell networking party, The Cyprus Institute of Neurology & Genetics

Dear delegates,

In light of the latest developments and as a precautionary measure, the Cyprus Presidency of the Council of the EU 2026 has decided to postpone this week’s Presidency Conference Advancement of Treatments for Rare Diseases, scheduled to take place in Cyprus 5–6 March 2026.

Further information regarding possible re-arrangement and new dates will be communicated in due course.

We apologise for the inconvenience caused by these adjustments and thank you for your understanding.

Yours sincerely,
The Organizing Committee
Conference “Advancement of Treatments for Rare Diseases”
Cyprus Presidency of the Council of the European Union 2026