Our Latest News
Keeping You Informed
September 2025
American Society for Bone and Mineral Research
The American Society for Bone and Mineral Research Conference showcased ground breaking clinical trials happening in Ireland, like Treatment of Osteogenesis Imperfecta with Teriparatide and Zoledronic acid trial (TOPaZ) and Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (COACH). Flying the flag were RDCTN co-lead Prof Rachel Crowley and Network members Liz Molloy, Prof Ciara McDonnell and Prof Stuart Ralston. Having such high calibre local research featured at a major US conference reflects Ireland’s strong credentials as a hub for developing future treatments!
September 2025
European Respiratory Society Congress 2025
RDCTN Research Data and Regulatory Affairs Officer, Sarah Forde, presented her work supporting research on metabolic pathways in Idiopathic Pulmonary Fibrosis through data support and analysis. Her poster was a great example of Irish rare disease research at one of the world’s largest meetings for education, innovation and discovery in lung health..
September 2025
HSE Patient and Public Partnership Conference 2025
RDCTN PPI Liaison Officer Dr Cassy Dinius and RDCTN PPI Partner Marci Kay Livingston attended the HSE Patient & Public Partnership conference in Croke Park. They presented a poster on the ‘many hats’ that PPI Partners wear, and how this enriches involvement and encourages an inclusive approach to rare disease research and clinical trials.
September 2025
RDCTN Seed Funding Call Open
We are delighted to launch the 3rd round of RDCTN seed funding to support emerging and early career researchers to build pilot data for developing an intervention and/or preparing for a clinical trial. We encourage applicants to include meaningful collaboration with PPI Partners in proposals. Read more
September 2025
Rare Disease Research – Early Career Guidance
Prof Carla Moran and Dr Donal O’Malley discuss essential skills and practical tips for a thriving career in rare disease research in this insightful and informative interview. For many patients with rare disease, the first and biggest challenge isn’t treatment, it’s a diagnosis. This video explores the vital role research plays in finding answers. Research is a diverse field and you don’t have to be a principal investigator to advance medicine – no matter your skill set, there’s a place for you!
Watch our full video here.
August 2025
Rare Disease Clinical Trial Success Story
Professor Cormac McCarthy and Linda Sheehan took part in an interview on RTE’s Clare Byrne Show to share the story of Linda’s diagnosis with a rare lung condition – autoimmune pulmonary alveolar proteinosis (autoimmune PAP) and her subsequent enrolment in the IMPALA-2 clinical trial. Linda described the transformative impact of taking part in the trial – from being unable to walk or stand without oxygen to being oxygen free and back working full-time. Prof McCarthy, who led the Irish arm of the RDCTN affiliated trial explained how this new inhaled therapy represents a ground-breaking shift in care for patients like Linda, offering real hope in a condition that often takes up to seven years to diagnose. Listen back to this inspirational interview here.
August 2025
National Rare Disease Strategy
We were delighted to attend the launch of Ireland’s new National Rare Disease Strategy. It is a hugely welcome milestone in improving the lives of people living with rare diseases across Ireland through enhanced diagnosis, treatment and support. We are especially encouraged by the strong focus on patient partnership and fostering research and innovation and to see RDCTN recognized as an advance in rare disease research in Ireland. We look forward to supporting its implementation.
August 2025
NEJM Publication of IMPALA 2 Findings
We are delighted to announce the publication of this RDCTN supported clinical trial, led in Ireland by RDCTN co-lead Associate Professor Cormac McCarthy. The trial found that daily inhaled molgramostim can significantly improve both the breathing function and quality of life for patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP). This study represents a major breakthrough in the treatment of this rare and debilitating lung disease.
July 2025
New Clinical Trials
We are delighted to announce the addition of three new clinical trials to our network. These are now open for the recruitment of patients with progressive and idiopathic pulmonary fibrosis.
Have you new rare disease trials in the pipeline? Please let us know – we can help raise awareness and support recruitment through our web and social media channels.
April 2025
Inclusive PPI Workshop
RDCTN and Health Research Charities Ireland co-hosted a workshop on creating inclusive spaces for PPI in rare disease research. It brought together researchers, charity representatives, and PPI contributors for panel discussions, themed breakouts, and open dialogue. Insights from the day will shape a practical guide for researchers, ensuring the rare disease community’s perspectives are meaningfully embedded in the design of future research.
April 2025
PPI Contributor Awards
RDCTN was delighted to have the opportunity to recognise the invaluable contribution of our PPI Panel members. Pictured are Gillian Stafford, Rachel Lynch and Stacey Grealis. Also recognised but not in the picture is Jackie Kiernan. Heartfelt thanks and appreciation for all that they do for rare disease research and clinical trials.
April 2025
Best PPI Poster Award
Congratulations to Eva Woods, TCD on being awarded the prize for Best PPI Poster at the Rare Disease Research Conference 2025.
The poster described a study on ‘Enhancing Huntington’s Disease Research through Public & Patient Involvement: A Collaborative Approach to Enhancing Research Priorities and Improving Research Outcomes.’
April 2025
Best Poster Award
Congratulations to Atia Battool, UCD on being awarded the prize for Best Poster at the Rare Disease Research Conference 2025.
The poster described a study on Defining the role of ATP-citrate lyase (ACLY) in Pulmonary Fibrosis Immunometabolism.
April 2025
RDCTN Seed Funding Award 2024/2025
Congratulations to Dr Carla Moran, Consultant Endocrinologist, recipient of the 2024 Award.
The project will explore Cardiac Phenotype of Resistance to Thyroid Hormone beta, a rare condition (1-2 people per 40,000) which is usually inherited.
April 2025
Rare Disease Research Conference 2025
Thanks to our speakers, moderators, poster presenters, sponsors, PPI partners and our wonderful delegates for contributing so richly to the success of our conference in O’Reilly Hall, UCD on the 10th April.
March 2025
ERN Lung News
Congratulations to our co-lead Prof Cormac McCarthy and his team on the naming of St Vincent’s University Hospital as Europe’s top-performing centre for rare lung disease by ERN Lung. It is a prime example of how participation in an ERN enables pioneering research, access to international expertise and a role in shaping the future of rare lung disease treatment and research.
March 2025
Use, Re-use and Sharing of Data Conference
RDCTN co-lead Prof Rachel Crowley shared insights from the work of RDCTN and RDCat in the rare disease research and clinical care settings. This provided valuable context for the discussion on evolving regulatory frameworks in Europe and ethical, legal & societal considerations for implementation of health data infrastructure and systems in Ireland.
February 2025
Why Research Matters
To mark World Rare Disease Day 2025 we highlight the value of research for people with a rare disease. Read this powerful insight into the many ways that research can make a difference in the words of Geraldine Halpin, a parent of an adult son with a rare disease and Cassandra Dinius, RDCTN PPI Liaison Officer. Click here
January 2025
Rare Disease Research Conference Abstract Submission
The Rare Disease Research Conference will take place on 10th April 2025 in the O’Reilly Hall UCD. A call for abstracts of rare disease case studies or research for poster presentation is now open – deadline extended to 24th February.
January 2025
Innovating with PPI
We are proud to share this engaging discussion led by Public and Patient Involvement (PPI) contributor Liz Molloy in conversation with researcher Prof Rachel Crowley exploring the impact and value of PPI in research.
January 2025
Research Tools Update
We continue to expand our Rare Disease Clinical Trial Toolbox with the addition of two new resources:
1. EuroGEMS.org – an ESHG-approved resource providing genetic and genomic information for educators and individuals.
2. Regulatory and Ethical Database (RED) by ECRIN.
DEcember 2024
PPI in RDCTN: How We Operate
In partnership with our Public and Patient Involvement (PPI) Panel we continue to develop our PPI structures and activities. As part of this we have co-created an information hub for people interested in learning more about how PPI in RDCTN operates.
December 2024
ERDERA’s first Joint Transnational Call is now open. The call aims to foster international collaborations to advance therapeutic solutions for rare diseases, benefiting millions of patients worldwide. Information is available from the Health Research Board.
December 2024
Partner Events
RDCTN team members were delighted to take part in two highly impactful events hosted by our partners in Health Research Charities Ireland – the Irish Health Research Forum on Public Trust in Health Research and the HRCI HRB Joint Funding Scheme Celebrations & Impact Award.
NOvember 2024
We were delighted to support UCD Rheumatology in the organisation of the Rare Rheumatology Conference – co-moderated by our PPI Liaison Officer Dr Cassandra Dinius and featuring a highly illustrative overview of rare bone cases by our Co-Lead, Prof Rachel Crowley .
october 2024
Research Tools Update
We are constantly looking out for resources to share with our research community. The latest additions to our Toolbox include an introduction to using RStudio compiled by our Research Data and Regulatory Affairs Officer Sarah Forde. We are also grateful to EJP-RD for kind permission to share their Virtual Platform Portal.
october 2024
Rare Disease PPI Panel
Calling PPI Contributors! Bring your perspective to our Rare Disease PPI Panel! With flexible time commitments and supports available, you can make a real impact by partnering on research that truly matters.
october 2024
We are excited to announce that the Rare Disease Research Conference will take place on the 10th April 2025 in the O’Reilly Hall, UCD. Topics will include Rare Disease Trial Methodologies, ERN Registries and Clinical Trial Case Studies. ‘Abstract call’ coming soon.
1st March 2024
RDCTN was delighted to present Dr Laura Williams, Consultant Neurologist, SVUH, with the 2023 Seed funding Award. Her project will pilot an inexpensive wearable device that measures dystonia severity to assess if it can offer a means to measure effectiveness of treatments in clinical trials and clinical practice. Find out more about funding opportunities here.
29th February 2024
RDCTN marked World Rare Disease Day 2024 with the inaugural Rare Disease Clinical Trials Conference. World leading researchers and patient advocates shared knowledge and experience across a range of topics. Find out more here.
28th June 2023
Prof Rachel Crowley (RDCTN co-lead) joined the Get Rare Aware Campaign lead by RDCTN Partner Vicky McGrath (Rare Diseases Ireland) and at the houses of the Oireachtas asking for improved genetic services in Ireland.
16th June 2023
Dr Siobhán Hendrick partnered with Health Research Charities Ireland to deliver a workshop on researchers and charities partnering at the PPI Ignite Summer School in University of Limerick.
14th June 2023
Dr Siobhán Hendrick was invited to present at the Rare Disease Forum delivering a talk on public and patient involvement in the Rare Disease Clinical Trial Network. The forum was hosted by RDCTN partners Health Research Charities Ireland.
25th May 2023
The Rare Disease Clinical Trial Network launched its first journal club aimed at building capacity in early career researchers.
17th May 2023
RDCTN Partners Vicky McGrath (Rare Diseases Ireland) and Prof Cormac McCarthy (RDCTN co-lead) participated in the Oireachtas cross party group meeting on rare diseases.
10th May 2023
RDCTN Partners Dr Derrick Mitchell (IPPOSI) Prof Rachel Crowley (RDCTN co- lead) and Prof Cormac McCarthy (RDCTN co- lead) participated in the Rare Disease Ireland (RDI) roundtable discussion.
25th Feb 2023
Prof Rachel Crowley was invited to deliver a talk at the Brittle Bone Society All Ireland Mini-Conference 2023.
Visit www.brittlebone.org
Training Opportunities
We are running an online Rare Disease Journal Club where we dive deep into scientific literature, and early career researchers have the opportunity to discuss a recent article in their own field. We will be announcing our next journal club soon. Join our mailing list to hear more!
PPI Opportunities
Do you have a lived experience of rare disease? Would you like to share your insights and experiences to help prioritise and shape clinical trials for rare diseases in Ireland? If so, we would love to hear from you. Email: rdctn@ucd.ie to express your interest.
Network Partners
