ERN-LUNG - European Reference Network for rare respiratory diseases

08/06/2026

The Hungarian Respiratory Society held its bi-annual meeting on 13-16 May in Balatonfüred, Hungary.

Thanks to the efforts of Prof. Ildiko Horvath, current Secretary General of the European Respiratory Society, a multistakeholder roundtable was organized to explore the difficulties of Alpha-1 Antitrypsin Deficiency (AATD) patients' access to medication in Hungary. ERN-Lung was also invited to this close-door negotiation.

As it turned out from the talks the problem is not solely linked to reimbursement hurdles (it is also existing), but the identification of these patients in Hungary: while there is a good understanding of the COPD population a bigger number of AATD patients should be detected based on international prevelance numbers.

The parties agreed that further work is needed and agreed that a follow-up meeting will be necessary.

28/05/2026

– Abstract selected for oral presentation at the American Thoracic Society International Conference 2026 –

May 23, 2026 – The international PCD Clinical Trial Network (PCD CTN) played a central role in the clinical ex*****on of a groundbreaking study investigating inhaled mRNA therapy in patients with primary ciliary dyskinesia (PCD), presented at the ATS 2026 International Conference in Orlando, USA (May 15–20, 2026).

Two highly specialized PCD CTN investigator sites were deeply involved in the study:

Danish PCD Centre, Copenhagen, Rigshospitalet (Caroline Nygaard, Tavs Qvist and Kim G. Nielsen)
University Children’s Hospital Muenster (Johanna Raidt and Heymut Omran)
In addition, a U.S. site at the University of North Carolina (UNC), Chapel Hill contributed to the trial.

The data represent the first clinical evidence of biological activity of a genetic medicine in PCD, a rare inherited disease characterized by progressive lung damage and the absence of disease-modifying treatments. These findings provide the first indication of successful delivery and translation of an inhaled mRNA therapeutic in the human airway.

Results from the Phase 1b trial (RCT1100-103), conducted across sites in Denmark, Germany, and the United States, showed that 57% of patients achieved a clinically meaningful improvement in mucociliary clearance (MCC) at 12 weeks. Bronchoscopy confirmed restoration of protein expression and ciliary function in the airways. This biological activity correlated with improvements in MCC. The therapy was safe and well tolerated, with no serious adverse events reported.

The study builds on earlier Phase 1 trials, which established the safety and tolerability of repeated dosing. In parallel, longitudinal observational data have demonstrated consistently reduced MCC across PCD patients regardless of genotype, supporting MCC as a robust endpoint for evaluating disease-modifying therapies in future studies.

These findings represent an important milestone for the PCD field and reflect a strong collaboration between international expert centers, patients, and industry partners. The investigational therapy was developed by ReCode Therapeutics, which contributed the underlying mRNA and lipid nanoparticle delivery technology.

About Primary Ciliary Dyskinesia (PCD)

Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterized by impaired mucociliary clearance (MCC), chronic respiratory infections, bronchiectasis, and progressive loss of lung function. Mutations in more than 50 genes lead to dysfunctional cilia and loss of MCC. PCD is a progressive disease with no curative or disease-modifying treatments currently available. It is estimated to affect at least 1 in 7,500 individuals, although up to 80% of patients are thought to remain undiagnosed.

About PCD CTN

The PCD Clinical Trial Network (PCD CTN) is an international network of specialized centers dedicated to advancing clinical research and therapeutic development for patients with primary ciliary dyskinesia. The network enables high-quality multicenter trials through standardized protocols, harmonized outcome measures, and close collaboration between clinicians, researchers, and patient organizations.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company developing mRNA and gene correction therapies using lipid nanoparticle-based delivery technologies for targeted treatment of disease.

19/05/2026

ERDERA invites the ERN community to an introductory webinar on “The ERDERA Diagnostic Research Workstream – An Overview”, taking place on Thursday, 28 May 2026, from 13:00 to 14:00 CEST.



Presented by Holm Graessner, this opening session will explain what the ERDERA Diagnostic Research Workstream does, how it is organised, and how clinicians, researchers and expert groups can contribute. The webinar will provide an overview of the workstream’s core activities, including data sharing, systematic diagnostic reanalysis, advanced diagnostic pipelines, and innovation in genomic and multi-omics technologies.



The session is particularly relevant for ERN healthcare professionals, geneticists, clinicians, JTC beneficiaries and anyone interested in diagnostic research.

Date: Thursday, 28 May 2026

Time: 13:00–14:00 CEST

Format: Online webinar

Find out more:

The session will introduce the workstream’s activities in data sharing, systematic diagnostic reanalysis, advanced diagnostic pipelines, and genomic and multi-omics innovation.

11/05/2026

We are working with the European Lung Foundation and European Respiratory Society to gather experiences of lung transplantation in Europe.

Two surveys have been launched as part of the ELF Chair’s Campaign, which aims to raise awareness, improve education and advocate for equal access to lung transplantation for people with severe lung conditions across Europe.

Patient carer survey
This is aimed at:
🫁 People who have received a lung transplant
🫁 People who have been referred, evaluated or are currently on the waiting list
💙 Family members and carers supporting someone through their transplant journey
🌍 Available in 13 languages with more on the way
🔗 https://europeanlung.org/en/get-involved/surveys/share-your-experience-of-lung-transplantation-in-europe/

➡️Please share this survey with patients in your network. You can share the link above directly, or share the flyers found in this folder: https://drive.google.com/drive/folders/1J2VDfbmeEAkAGTbkIM-hx04b-vr6L2ph?usp=sharing

Transplant centre survey
To help us build a clearer picture of current practice, identify gaps and inequalities and support future advocacy, education, and service improvement. If you are a representative of a transplant centre, please complete the survey below available in English: https://www.surveymonkey.com/r/RVQHB8J

By sharing your experience, you will help us build a clearer picture of lung transplantation across Europe, identify gaps and inequalities and strengthen future advocacy and service improvement.

Survey open until 18th May 2026.

20/04/2026

European Parliament draft report urges a binding EU rare disease action framework

A draft report from the European Parliament’s Committee on Public Health (SANT), spearheaded by MEP Nicolás González Casares, urges the European Commission to introduce a binding legislative framework for rare diseases. The proposal aims to address existing fragmentation and inequalities in diagnosis, treatment, research, and data infrastructure. Structured similarly to an EU Regulation, the framework comprises six pillars and advocates for centralized coordination, standardized indicators, and mandatory reporting. Additionally, the report seeks to strengthen European Reference Networks (ERNs) by improving their governance and ensuring their long-term integration into national healthcare systems.
Find out more: https://erdera.org/news/european-parliament-draft-report-urges-a-binding-eu-rare-disease-action-framework/?utm_source=Sarbacane&utm_medium=email&utm_campaign=ERDERA%20NEWSLETTER%20 #6

A legislative own initiative file in the Parliament’s new public health committee sets out what an EU “rare disease action framework” could look like — and why it could change how Europe measures progress for patients.

17/04/2026

2026 Call for Applications – Affiliated Partner within ERNs

Eligible Countries: Bulgaria, Cyprus, Croatia, Czechia, Estonia, Greece, Ireland, Latvia, Norway, Poland, Portugal, Romania, Slovenia, and Slovakia.
Application Limit: Eligible countries can submit up to three applications per missing ERN.
Key Objectives: Expand Coverage: Improve the geographic reach of ERN expertise across the EU and Norway.
Increase Access: Provide specialized care in regions or disease areas where full ERN membership is currently missing.
System Integration: Better integrate ERNs into national healthcare systems.
Knowledge Sharing: Support cross-border clinical collaboration and exchange.
Opening date: 13 April 2026
Deadline: 1 September 2026
Find out more:

2026 call for applications

25/03/2026

25/03/2026

Stephy Varghese T.'s presentation at ERN-LUNG's Annual Board Meeting (ABM) held in Paris offered a thoughtful reflection on how the meeting itself has evolved over the years. She focused on how the meetings have grown into a dynamic platform for strategic dialogue, cross-functional alignment, and long-term vision setting, highlighting how the board meeting has increasingly become a space not just to review results, but to challenge perspectives, foster collaboration, and shape the future direction of ERN-LUNG. Her insights underscored the value of these moments in bringing leadership together with clarity and purpose - making the annual board meeting not just a milestone event, but a key driver of ERN-LUNG's continued growth and transformation.

25/03/2026

At the recent ABM in Paris, Donata Eick had the opportunity to present on behalf of ERN-LUNG, highlighting the importance of transition and the continued need to build momentum across the network.

The session sparked strong engagement and reinforced a shared commitment to driving this work forward together. Donata also showcased key quality improvements achieved by ERN-LUNG over the past five years—demonstrating the network’s ongoing dedication to advancing care for patients with rare respiratory diseases.

An important part of the meeting was the discussion with members, where priorities for the next phase were defined through a collective vote. The agreed focus areas are:
- Advancing AI in rare diseases
- Strengthening collaboration on an Inter ERN scale
- ⁠continuing to expand an make patients the center of the network.

ERN LUNG remains committed to turning these priorities into meaningful action and impact for patients, clinicians, and the wider community.

25/03/2026

Gergely Meszaros JD, MSc presented the results of the Go East project, and also covered some aspects of the methodology. We discussed how we can overcome some of the difficulties as in a couple of cases on the last mile is missing to onboard an additional healthcare provider to our network.