ERDERA has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595.
Views and opinions expressed are those of the author(s) only and do not necessarily reflect those of the European Union or any other granting authority, who cannot be held responsible for them.
© 2025 | ERDERA — European Rare Diseases Research Alliance
ERDERA is coordinated by Insem
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have jointly issued a clear warning about unregulated Advanced Therapy Medicinal Products (ATMPs). While these therapies—based on genes, tissues or cells—offer genuine promise, its limited regulation procedures raise serious health and safety concerns. Authorities urge patients to confirm that any ATMP is approved through official channels to avoid potential risks.
We met Irene Norstedt, Director for the People Directorate at DG Research and Innovation in the European Commission, at the RE(ACT) Congress in Brussels, where she delivered a compelling presentation on diverse funding models targeting different research needs.
The 8th RE(ACT) Congress and the 6th IRDiRC (International Rare Diseases Research Consortium) Conference concluded today, marking the end of a three-day summit where scientists, patient advocates, funders, and policymakers tackled the rare disease field’s most pressing challenges.
As Rare Disease Day 2025 comes to a close, its impact continues to resonate across Europe and beyond. Held annually on the last day of February, this year’s event drew critical attention to the 30 million people in Europe living with a rare disease, underscoring the urgent need for faster diagnoses, increased research, improved treatments, and stronger policies.
On 28 February, coinciding with the 2025 Rare Disease Day, Parliament’s public health committee, SANT, launched an online survey to gather insights on rare diseases. The initiative aims to build a detailed understanding of the challenges faced by individuals affected by rare diseases, as well as to collect views from stakeholders—including patients, caregivers, industry representatives, and NGOs—to inform future policy and legislative reforms.
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have jointly issued a clear warning about unregulated Advanced Therapy Medicinal Products (ATMPs). While these therapies—based on genes, tissues or cells—offer genuine promise, its limited regulation procedures raise serious health and safety concerns. Authorities urge patients to confirm that any ATMP is approved READ MORE
We met Irene Norstedt, Director for the People Directorate at DG Research and Innovation in the European Commission, at the RE(ACT) Congress in Brussels, where she delivered a compelling presentation on diverse funding models targeting different research needs. READ MORE
The 8th RE(ACT) Congress and the 6th IRDiRC (International Rare Diseases Research Consortium) Conference concluded today, marking the end of a three-day summit where scientists, patient advocates, funders, and policymakers tackled the rare disease field’s most pressing challenges. READ MORE
As Rare Disease Day 2025 comes to a close, its impact continues to resonate across Europe and beyond. Held annually on the last day of February, this year’s event drew critical attention to the 30 million people in Europe living with a rare disease, underscoring the urgent need for faster diagnoses, increased research, improved treatments, READ MORE
On 28 February, coinciding with the 2025 Rare Disease Day, Parliament’s public health committee, SANT, launched an online survey to gather insights on rare diseases. The initiative aims to build a detailed understanding of the challenges faced by individuals affected by rare diseases, as well as to collect views from stakeholders—including patients, caregivers, industry representatives, READ MORE
Imagine having a disease so rare that doctors struggle to diagnose it, researchers have little data to study it, and treatments are scarce. For the 30 million people in Europe living with a rare disease, this is a daily reality. But change is happening. READ MORE
The landscape of clinical research and genomic data governance is evolving rapidly, with two significant developments shaping the future of global health research and regulatory frameworks: the ongoing revision of the ICH E6 (R3) Good Clinical Practice (GCP) guideline and the recent release of WHO guidance on the ethical collection, access, use, and sharing of READ MORE
We are pleased to announce that the platform for submitting project applications for the Joint Transnational Call for Proposals (JTC) 2025 is now officially live. This highly anticipated call invites research teams across Europe and internationally to collaborate on cutting-edge projects aimed at “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – READ MORE
