ECRD 2024
2024-05-15 08:00 - 2024-05-16 17:00 (Europe/London) | The Square Venue, Brussels
The 12th European Conference on Rare Diseases & Orphan Products (ECRD) is recognised globally as the largest, patient-led, rare disease policy-shaping event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.
Leading, inspiring and engaging all stakeholders to take action, the Conference is an unrivalled opportunity to network and exchange invaluable knowledge with over 1000 stakeholders in the rare disease community – patient advocates, policy makers, researchers, clinicians, healthcare
professionals, healthcare industry representatives, academics, payers, regulators and Member State representatives.
With the European elections approaching, at the inception of a new term for the European Parliament and the European Commission, the 12th ECRD becomes a key driver in shaping policies to address the unique needs of people with rare diseases and their families over the next
five years.
The ECRD’s value is underscored by its alignment with EURORDIS’ priorities for the EU elections, evident in its comprehensive six-session agenda.
What sets the ECRD apart is its integration into broader rare disease policy and healthcare ecosystem efforts. Rooted in the recommendations of the Rare 2030 Foresight Study, the ECRD continues to build on this comprehensive review of the strategy for rare diseases and shape the thinking of policymakers and the community, encouraging a more streamlined and proactive approach to addressing the unmet needs of people living with rare diseases and the persisting inequalities across Europe.
Save the Date: 15 – 16 May 2024, online and at The Square venue in Brussels

Misdiagnosis and surgical uncertainty - A patient's perspective
2024-09-25 19:30 - 21:00
Charlotte Cuffe is a CAUK member and her patient story was recently featured in a prestigious global essay competition after she was interviewed by 5th Year Medical Student, Iman Muzafar. Join us to hear Charlotte and Iman in conversation about misdiagnosis and surgical uncertainty. To read Iman’s award-winning essay published in Springer Nature go to https://communities.springernature.com/posts/student-voice-prize-runner-up
This is the registration link for the event: https://us06web.zoom.us/webinar/register/WN_GNz8TK1lQN-wUqrXDtaXKQ#/registration

Rare Fest 2024
2024-11-22 08:00 - 2024-11-23 17:00
RAREfest24
A festival as unique as you are. For curious minds of all ages, for FREE.
Fri 22 November – Launch Evening ‘Strictly Rare’
An evening of music, performance, comedy, talks and innovative research inspired art to challenge perceptions, dispel myths and imagine the possible.
Sat 23 November – Interactive Exhibition
Immerse yourself in a bustling marketplace of hands-on exhibits, interactive talks, films, and stunning research-inspired art. Discover the latest in innovative science and technology and meet leading researchers, tech start-ups, treatment pioneers and individuals with rare conditions. Educational fun for the whole family.

Haematology
2024-11-22 11:00 - 12:30
Alongside the British Society for Haematology this session will discuss management of sickle cell disease and the appropriate use of blood components. It will also explore diagnostic work up for suspected haematological malignancy and investigate thrombocytopenia.

Connecting the Dots: Oxford University Student Rare Disease Conference 2025
2025-02-23 09:30 - 17:00 | Magdalen College, University of Oxford
The Oxford Rare Disease Society (OxRare) and the Sherrington Society are excited to welcome you to “Connecting the Dots” – our first Oxford University Student Rare Disease Conference, taking place Sunday 23rd February, 9:30–17:00 at Magdalen College, Oxford. We’re supported by Medics for Rare Disease and our kind sponsors.
Join us for this exciting opportunity to hear from expert speakers at the frontiers of genomics and medical research, amplify Rare Disease patient voices, forge new collaborations, and learn how we can make a positive difference as the doctors and science professionals of tomorrow.
Call for posters! Students and early career researchers are invited to submit a poster presentation, with chance to win prizes! Submit your abstracts here: https://forms.gle/D7VBjtYKbQagQmk97
In-person ticket includes FREE tea, coffee, delicious lunch and networking drinks. We just ask that you complete the feedback form to receive your Certificate of Attendance and part-reimbursement of the ticket cost. The event is FREE to attend for individuals with lived experience of rare disease.
NB: Livestream tickets are intended for those joining virtually from outside of Oxford. Those in Oxford are highly encouraged to attend in person to enjoy free refreshments and the opportunity to speak to Rare Disease patient representatives.
Any questions you can email Linda/Kevin at oxfordrarediseasesoc@gmail.com or Cat/Jake at magdsherringtonsociety@gmail.com.
Getting here:
The closest entry to the Grove Auditorium is through the entrance on Longwall Street (step-free access). Alternatively, if you enter via the Porters’ Lodge on the main High Street (non step-free), they can help to direct you to the Auditorium.
By Public Transport: From Oxford Railway Station, Magdalen College is ~20 min walk, ~20 min by bus, and 10 min by taxi. The nearest bus stop for the college is High Street (2 min walk), served by buses from the Railway station and by the Oxford Tube.
By Car: You may wish to use one of the Park & Rides. The fare includes parking for 16 hours and bus travel into Oxford City Centre https://www.oxford.gov.uk/park-ride

More Than You Can Imagine: Genomics and Rare Disease
2025-02-28 12:00 - 14:30 | ONLINE
Join Central and South Genomics for an insightful webinar in recognition of Rare Disease Day, where we bring together patients, healthcare professionals, and researchers to explore the latest in patient advocacy, genomics and rare disease. This event is for everyone, whether you’re from a healthcare background, have been affected by a rare disease, or are just keen to learn more.
This session will cover key topics, including:
Understanding rare diseases and genomics – How advances in genetics are shaping diagnosis and treatment.
Equity & diversity in research – Addressing challenges in representation and access to care.
Patient experiences – Real-life stories that highlight the journey from diagnosis to treatment.
Newborn research & pharmacogenomics – The future of personalised medicine.
Support networks & advocacy – Connecting patients with resources for ongoing care.
We’ll hear from a range of experts, from clinicians to charity representatives and patient advocates. To view the full agenda, please click here.
If you have any queries, please don’t hesitate to get in touch. We look forward to seeing you on 28 February.

Swansea Rare Disease Study Day
2025-03-08 10:00 - 18:30 | Morriston Hospital Education Centre, Heol Maes Eglwys Treforys SA6 6NL
Dive deep into the world of rare diseases at the first Swansea Rare Disease Study Day on 8th March 2025!
Why attend?
Gain invaluable insights from our expert guest speakers.
Learn from our engaging panel discussions.
Meet a range of local patient advocacy groups to discover more about the lived reality of those impacted by rare conditions.
Network with others rare disease allies.
Enjoy a tasty lunch from Sue Franks catering.
Call for oral and poster presentations
Medical students and foundation doctors are invited to submit abstracts for oral presentations, and medical students and resident doctors of all grades for poster presentations. Cash prizes are up for grabs! Find out more and submit your abstract here https://forms.gle/pFhybxbJo1XbPU5U9 by 31st January 2025.
Don’t miss this opportunity to expand your horizons and become #RareAware!

Soft UK Healthcare Professionals Seminar
2025-05-16 14:00 - 16:00 | Holiday Inn, Birmingham
SOFT UK invites healthcare professionals to a transformative seminar focused on the power of language in supporting families navigating rare genetic diagnoses and ongoing care. This event offers a unique blend of expert insights and heartfelt personal stories, equipping attendees with practical tools to foster meaningful and compassionate dialogue.
By attending, you will:
Develop your understanding of the impact of language upon parents and families facing diagnosis of rare genetic and life limiting conditions
Engage in face-to-face networking with trisomy family members, gaining first hand perspectives
Discover actionable strategies through real-life narratives to enhance communication during diagnosis and throughout ongoing care
At the end of the event we invite you to join us at a Rewards and Recognition dinner, where you can learn more about the work of SOFT UK and those who support us.

Genomics in Primary Care: What GPs need to know
2025-06-12 12:00 - 14:00 | ONLINE
Genomic medicine has a significant impact within the field of primary care. Generally acting as the gateway to the NHS and specialised services, primary care practitioners are crucial in the early identification of genetic conditions and in ensuring appropriate management of a range of conditions.
Genomics features in everyday primary care practice in a range of ways:
* Rare diseases are individually rare but collectively affect 1:17 families. GPs may have a caseload with a number of patients with the same rare disease within a family.
* Most care for patients with genetic conditions is provided in the community. A patient’s genetic condition might influence your choice of commonly prescribed medications such as HRT or contraceptives.
* Some common conditions have genetic susceptibility. Clinical Genetics only see the patients that GPs refer to them. As a GP you are in a unique position to suspect these diagnoses early and refer appropriately.
* Patients with inherited cancer predisposition syndromes need regular screening to find cancer early and improve prognosis. You can encourage them to attend for this.
* GPs can give preconception advice for families with known genetic conditions and encourage relatives to be tested to benefit from additional screening/treatment.
* Genes influence the way that our bodies process medication. As more research emerges, genetics is likely to influence our approach to prescribing in the future. This is already being seen in the prescription of clopidogrel to patients who have had a stroke.
In this informative webinar, we’ll hear from a range of experts – from patients through to healthcare professionals – to examine the application of genomics within primary care and how it relates to your role.
Register now to secure your place here, and don’t hesitate to get in touch if you have any questions.

Recommendation Writing Workshop
2025-06-23 09:30 - 16:00 | Edgbaston Park Hotel
Monday 23rd June 9.30 – 4pm at Edgbaston Park Hotel. LIMITED PLACES – reserve your spot now!
Welcome to the IAG2 Face to Face Meeting!
Medics for Rare Disease has a vision of healthcare equity for everyone. We have been working with the Department of Health and Social Care since 2018 in order to achieve our mission.
On 23rd June we are hosting a workshop in order to develop recommendations for the DoHSC about ‘healthcare professional awareness of rare disease’.
We will be asking…
What is healthcare professional awareness?
How do we implement it throughout the NHS?
How do we measure it?
If you have practical ideas and suggestions that could inform these recommendations, WE WANT YOU to join us on the day.
Who do we want to attend:
People who have practical ideas
People who have experience of driving system change
People who have experience developing services in the NHS
People with experience designing and implementing e.g. Dementia Friendly Hospitals
Healthcare professionals, people who work in the NHS and advocates alike!
This project (‘IAG2’) is being run by Medics for Rare Disease and Emotive with support from collaborators on the UK Rare Diseases Framework Forum and has been approved by The Department of Health and Social Care.
Keep in mind:
We are not responsible for implementing the recommendations
This will not be a forum for exploring single individual’s experiences
This will not be a forum for raising concerns or complaints about single cases of care
Medics for Rare Disease – www.m4rd.org
Emotive – https://emotiveagency.com
Reserve your place here

Our Voices, Our Stories: Lived Experiences of Genomic Testing
2025-07-15 12:00 - 14:00 | ONLINE
Genomic testing is a complex clinical topic, with new developments and discoveries taking place at a rapid rate. Often, we focus predominantly on the clinical and scientific aspects of genomics, but it’s crucial to remember that each advancement has life-changing impacts on people across the world.
In this webinar, we’ll be exploring the lived experiences of people who have gone through genomic testing, research and diagnosis. Each of the participants you will hear from has a unique and impactful story, and will provide powerful insights into the human side of the genomic testing process. With experiences across cancer and rare disease, from parent carers through to those diagnosed as adults, we’ll be shining a light on the emotions involved in testing, diagnosis and treatment. Not only will you get to hear these compelling stories – you’ll also have the opportunity to ask questions and learn more about the importance of patient and public involvement in genomic testing and research.
The webinar will be co-chaired by two clinical experts in genomics – Dr Hannah Titheradge, Consultant Clinical Geneticist at Birmingham Women’s and Children’s Hospital (BWC), and Emma Douglas, Genetics Clinical Research Practitioner at BWC. Hannah has years of experience working in clinical genetics and is the Rare Disease Lead at BWC, whilst Emma is an expert in genetic research and is currently working towards her PhD on the patient experience of genomic testing.
This session is open to everyone, whether you’re a patient, healthcare professional, or an interested member of the public. If you’ve got lived experience yourself or any questions, you are welcome to share during the Q&A. For healthcare professionals, the session promises to provide you with a better understanding of the crucial patient perspective to better support those in your clinic.
If you have any queries about the event, please get in touch. If you’ve got any accessibility requirements, please let us know during the registration process.
And if you’re interested but aren’t able to attend on the date itself, make sure to still register and we will send you the recording after the event has taken place!
You can sign up here

Introduction to Delphi Studies
2025-07-16 15:00 - 16:00 | ONLINE
Have you ever heard of a Delphi Study and not quite sure what it entails? Or maybe you have never heard of Delphi? This webinar will be providing training on what a Delphi Study is and how they are run. PhD Candidate, Shams Al-Ani, studying Pharmaceutical medicine and regulatory affairs at Centre for Pharmaceutical Medicine Research, King’s College London will be delivering the training through a 45 minute presentation with time for Q+A session afterwards.
Sign up here

RAREsummit25: CamRARE at 10 – Connecting Moments
2025-11-06 09:00 - 17:00 | Wellcome Genome Campus Conference Centre
2025 marks a special milestone — CamRARE’s 10th anniversary. A decade of progress. A future of possibilities. Over the past decade, we have championed collaboration, amplified patient voices, and driven progress in rare disease research, policy, and innovation. Through inspiring keynotes, dynamic discussions and workshops, and interactive networking opportunities, we will reflect on the past 10 years of impact while shaping the future of rare disease collaboration.
Who will you connect with at RAREsummit25?
Being connected is critical in rare diseases and RAREsummit25 brings the right people together – thought leaders, researchers, healthcare professionals, industry pioneers, policy-makers and —most importantly—individuals living with rare conditions and the organisations that support and advocate for them.
You can book tickets for the event here

Demystifying the connection between patients and researchers
2026-03-06 12:00 - 17:00 | Kings College London, Great Hall, King’s College London – Strand Campus, London, London, WC2R 2LS, United Kingdom
Co-hosted by Medics for Rare Disease and King’s Health Partners Rare Disease Network
📅 Date: Friday 6 March 2026
⏰ Time: 12:00 – 16:45
📍 Location: Great Hall, King’s College London – Strand Campus, WC2R 2LS (In-person event)
About the event
Medics for Rare Disease is pleased to be co-hosting a half-day, in-person event with the King’s Health Partners Rare Disease Network.
Join us for a half‑day event exploring how patients, researchers, clinicians and industry partners can work together to drive inclusive, impactful rare disease research.
Hear real stories, take part in discussions, and connect with others working to build equitable research partnerships.
Programme highlights
* Presentations
* High tea reception with poster displays (more details about Call For Abstracts coming soon)
* Expert panel discussions.
Who should attend
This event will be of interest to:
Patients and patient advocates
* Researchers and clinicians
* Industry partners
* Policy makers and research funders
* Anyone interested in inclusive, collaborative rare disease research
Registration
Buy your tickets here

European Conference on Rare Diseases & Orphan Products (ECRD 2026)
2026-06-02 - 2026-06-04 | O2 universum Congress Centre, Českomoravská 2345/17, Libeň, Prague, 19000, Czech Republic
🗓 Date: 2–4 June 2026
📍 Location: Prague & Online
🔗 Register: Sign up here
Join EURORDIS‑Rare Diseases Europe for the largest patient‑led rare disease policy event in Europe! Over three days, patients, advocates, clinicians, researchers, and policymakers will come together to discuss therapy access, diagnosis, care, innovation, and more.
We’re proud to support EURORDIS in sharing this opportunity and encouraging participation across our networks.
➡️ Register now: https://www.rare-diseases.eu/register/

2nd European Congress on Rare Diseases & Orphan Drugs
2026-09-14 - 2026-09-15 | Renaissance London Heathrow Hotel, London, United Kingdom
The 2nd European Congress on Rare Diseases & Orphan Drugs, organised by C2P Forum, brings together researchers, clinicians, industry leaders, policymakers and patient advocates to explore the latest advances in rare disease research, diagnosis, treatment and orphan drug development. The two-day programme includes keynote presentations, panel discussions, workshops, networking opportunities and an exhibition showcasing emerging technologies and therapies. The congress aims to encourage collaboration and accelerate progress towards improving outcomes for people living with rare diseases.
Who should attend?
* Healthcare professionals
* Researchers and academics
* Pharmaceutical and biotech professionals
* Patient advocacy organisations
* Policymakers
* Anyone with an interest in rare diseases and orphan drug development
Find out more and register
Visit the official event website:
2nd European Congress on Rare Diseases & Orphan Drugs

RAREsummit26
2026-10-07 | Wellcome Genome Campus Conference Centre
RAREsummit26 (Partner Event)
📅 Date: Wednesday 7 October 2026
📍 Location: Wellcome Genome Campus, Cambridge (with hybrid livestream option)
We’re delighted to share RAREsummit26, hosted by our friends at CamRARE (Cambridge Rare Disease Network).
Bringing together patients, advocates, clinicians, researchers, industry and policymakers, RAREsummit26 is a unique opportunity to make new connections, exchange ideas and explore how collaboration can accelerate progress for people living with rare diseases.
This year’s summit features:
* Inspiring keynote presentations
* Panel discussions and interactive workshops
* Networking opportunities
* Research posters and exhibition space
* Hybrid attendance options for those joining remotely
Whether you’re working in healthcare, research, patient advocacy or the wider rare disease community, RAREsummit26 offers an excellent opportunity to learn, connect and collaborate.

World Orphan Drug Congress Europe 2026
2026-10-26 - 2026-10-28 | RAI Congress Centre, Amsterdam, Amsterdam, Netherlands
The World Orphan Drug Congress Europe returns to Amsterdam from 26–28 October 2026, bringing together the global rare disease and orphan drug community for three days of collaboration, innovation and knowledge sharing.
Recognised as one of the world’s leading rare disease conferences, the event welcomes patient organisations, researchers, clinicians, regulators, policymakers, pharmaceutical and biotech companies, investors and industry leaders. The programme covers the full orphan drug development pathway, including cell and gene therapies, genetic testing, clinical trials, market access, regulatory affairs, real-world evidence and patient engagement.
Pre-congress workshops take place on 26 October, followed by the main congress on 27–28 October at the RAI Congress Centre, Amsterdam. The event provides valuable opportunities to learn about the latest advances in rare disease research, connect with international experts and explore innovative approaches to improving diagnosis, treatment and access to therapies.
Location: RAI Congress Centre, Amsterdam, Netherlands
Dates: 26–28 October 2026
Format: In-person
