News & Updates

EMA invites feedback to strengthen how the EU generates and uses evidence that reflects what matters to patients, including in rare diseases
Accelerating evidence-driven innovation to unlock fresh indications for established therapies.
A successful training event with 350 registrations from 58 countries and daily attendance averaging 160 participants
Oct
27
- 28
Oct
Monday,
All day
On-site event
No Venue Found

We’re pleased to announce that ERDERA will be attending #WODC2025 in Amsterdam on 27–28 October, driving collaboration and advancing research in the field of rare diseases. Come and visit us […]

Oct
30
Thursday,
09:00 am-11:20 pm
Online event
No Venue Found

ERDERA will mark its 1st anniversary with a stimulating online event open to the entire rare disease community. Leading experts in the field of neurology, neurometabolic diseases and patient advocacy […]

Nov
03
- 04
Nov
Monday,
All day
Online event
No Venue Found

The course will focus on the current status and advancements of clinical research especially to address unmet medical needs. Innovative methodologies, regulatory challenges, and the involvement of adolescents in clinical trials will be discussed.

Nov
14
- 16
Nov
Friday,
All day
On-site event
Izmir

Bringing together global experts, healthcare professionals, patient advocates, and policymakers to advance knowledge and strengthen international collaboration in the field of rare diseases.

Dec
09
- 10
Dec
Tuesday,
All day
On-site event
No Venue Found

ERDERA – the European Rare Diseases Research Alliance – is pleased to announce the upcoming 2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases, co-organised in collaboration with […]

Significant health inequities pose challenges for those living with a rare disease
Tandem Mass Spectrometry (MS/MS) has revolutionised newborn screening by enabling the simultaneous detection of dozens of metabolic and genetic disorders from a single dried blood spot. This powerful analytical technique has dramatically expanded screening capabilities since its introduction in the late 1990s.
Regulatory sandboxes have emerged as an innovative mechanism to facilitate the development and approval of new technologies, including pharmaceuticals. Their application in the context of rare disease therapies presents a promising avenue to accelerate the development, approval, and access to disease-modifying and life-saving therapies. Given the complexities of rare diseases and the regulatory hurdles faced by orphan medicinal products, regulatory sandboxes offer a structured yet flexible environment where new regulatory approaches can be tested and refined.
Complex regional pain syndrome (CRPS) is a rare, disabling pain disorder. Systematic reviews have identified a critical lack of high-quality clinical trial evidence to inform the management of CRPS. There is an urgent need to find solutions to the methodological challenges of undertaking clinical trials in CRPS. The aim of this project was to develop a methodological framework for optimising future clinical trials in CRPS (OptiMeth-CRPS).
This practical “Short Guide on Patient Partnerships in Rare Disease Research Projects” was created by the European Joint Programme on Rare Diseases (EJP RD) to show scientists, funders and patient organisations how to build meaningful, two-way collaborations in basic, pre-clinical, translational and social-science studies.

🗣 Throughout the week leading to Rare Disease Day 2025, we will be sharing insights from diverse voices highlighting key pathways to improving the lives of those affected. ️

Tomasz Grybek, carer, patient advocate, and Member Board of Directors of EURORDIS – a remarkable alliance of over 1,000 rare disease patient organisations from 74 countries – reminds us of the incredible strength and unity within the rare disease community.

Understanding the Ethics Self-Assessment Table for Networking Support Scheme Proposals 🧩 Are you preparing a proposal for the Networking Support Scheme?

This tutorial walks you through how to accurately complete the Ethics Self-Assessment Table, adapted from the Horizon Europe template.

Completing Section 9 for any other ethical issues relevant to your proposal ✅ Whether you’re new to the application process or just need a refresher, this step-by-step guide will help ensure you meet the ethical requirements of your submission. 📌 Don’t forget to like, share, and subscribe for more guidance on EU funding calls and application processes.

The #Together4RD Toolkit is a new strategic resource to support effective, transparent, and impactful ERN-industry collaborations in #RareDisease research. Developed with input from both #ERN and industry, the Toolkit offers practical guidance, case studies, and tools to tackle legal, structural, and practical barriers to public-private partnerships.

🗣 Throughout the week leading to Rare Disease Day 2025, we will be sharing insights from diverse voices highlighting key pathways to improving the lives of those affected. ️

In this video, Dorica Dan, rare disease carer and EURORDIS-Rare Diseases Europe Vice-Director, highlights the profound importance of early diagnosis for people living with a rare disease and their families – even when no treatment is available. 🔍

🗣 Throughout the week leading to Rare Disease Day 2025, we will be sharing insights from diverse voices highlighting key pathways to improving the lives of those affected. ️

In this video, Alexandre Méjat, a rare disease patient, PhD scientist in the field of rare diseases, and EURORDIS Director, shares his invaluable insight on the vital role of data collection in achieving quicker diagnoses. 📊⏱

To leave no one behind, over 170 organisations championed by the European Union and members states are working hand in hand to make Europe a world leader in rare diseases research and innovation. The European Rare Disease Research Alliance (ERDERA) takes over EJPRD to deliver concrete health benefits to rare disease patients in the next decade by advancing prevention, diagnosis and treatment research.