2025 Black Pearl Awards
On 24 February 2025
At : Brussels, Belgium (hybrid)
Events
RDI webinar on the WHA Resolution on rare diseases
On 27 February 2025
At : Online
Rare Disease Day 2025
On 28 February 2025
At : Worldwide
EURORDIS Rare Disease Day event at EU Parliament
On 05 March 2025
At : Brussels, Belgium
RE(ACT) Congress and IRDiRC Conference 2025
From 05 to 07 March 2025
At : Brussels, Belgium
13th ISNS European Regional Meeting
From 23 to 26 March 2025
At : Luxembourg
4th International Consensus Meeting on Robin Sequence
From 03 to 04 April 2025
At : Paris, France
2nd ERN ReCONNET International Congress on Rare and Low-Prevalence Connective Tissue Diseases
From 09 to 11 April 2025
At : Prague, Czech Republic
Endo-ERN General Assembly 2025
From 09 to 10 May 2025
At : Copenhagen, Denmark
International Primary Immunodeficiencies Congress 2025
From 05 to 07 November 2025
At : Prague, Czech Republic
Courses and Educational Initiatives
Upcoming ERN webinars
The European Reference Networks often organise educational webinars and other online meetings for their members and/or other interested parties. Below is a list of some upcoming events that may be of interest to our readers:
- 13/02 - EURO-NMD webinar on Multifocal motor neuropathy (MMN)
- 20/02 - EURO-NMD webinar on Electrodiagnostic patterns in immune-mediated neuropathies
- 11/03 - ERKNet/ERA webinar on FSGS in adults
- 17/03 - Endo-ERN webinar on Long-term outcomes in CAIS
- 20/03 - EURO-NMD webinar on Peripheral nerve imaging with focus on inflammatory neuropathies
- 27/03 - EURO-NMD webinar on Navigating life with dysimmune and inflammatory neuropathies (Q&A session on quality of life and management)
- 08/04 - TransplantChild webinar on organ xenotransplantation in children: dream or reality?
- 22/04 - TransplantChild webinar on impact and outcomes after optimization of liver bipartition in pediatric liver transplantation
- 29/04 - ERKNet webinar: Consensus statement on the diagnosis and management of CNDI
- 06/05 - ERKNet & MetabERN webinar on lysinuric protein intolerance
- 13/05 - TransplantChild webinar on special considerations for intestinal or combined liver-intestine transplantation in infants
- 20/05 - TransplantChild webinar on liver heart axis
- 10/06 - TransplantChild webinar on stem cell transplantation in paediatric AML
- 10/06 - ERKNet webinar on clinical practice recommendations on kidney management in methylmalonic acidemia
- 10/06 - TransplantChild webinar on biliary complications after paediatric liver transplantation
- 08/07 - TransplantChild webinar on challenges in managing persistent ascites post-liver transplantation
- 09/09 - TransplantChild webinar on portal flow modulation in paediatric liver transplantation
- 09/09 - TransplantChild webinar on volumetric absorptive microsampling (VAMS) as a novel toul for TDM samples collection in paediatric transplant recipients
- 16/09 - TransplantChild webinar on neonatal organ donation
ERN-EYE elearning course on inherited retinal diseases
The ERN-EYE has launched a second year of their elearning course on inherited retinal diseases. This course, which is aimed at medical professionals, features 30 hours of synchronous and asynchronous online teaching, delivering a deep understanding of the genetic foundations and clinical presentations of these conditions. Registration is free and open until 14 March 2025.
IDEA4RC webinar series
The EU-funded project IDEA4RC (Intelligent ecosystem to improve the governance, the sharing and the re-use of health data for rare cancers) is organising a series of upcoming webinars:
- 19/2 - Automatising data extraction and securing data storage: the data flow in IDEA4RC
- 26/03 - The "European Health Data Space" (EHDS): the new EU regulation on the re-use of health data
- 28/04 - Data altruism: principles and IDEA4RC solutions to enable data donation
RDI public webinar
On 12 February 2025
At : Online
Rare Diseases International (RDI) will be holding a public webinar on 12 February 2025 to present the results of their recent survey on rare disease expertise, care-providing centers and networks.
Cross-ERN online paediatric colorectal course
From 13 to 14 February 2025
At : Online
From 13-14 February 2025, the ERNs eUROGEN and ERNICA will be holding an online paediatric colorectal course, in collaboration with the European Paediatric Surgeon's Association (EUPSA) and ARM-Net. This two-day immersive program will focus on anorectal malformations (ARM) and Hirschprung's disease, offering hands-on training that can be completed from home.
6th ERN-RND Winter School
From 12 to 14 March 2025
At : Online
The ERN-RND and the European Pediatric Neurology Society (EPNS) are organising the 6th edition of their Winter School from 12-14 March 2025, focusing on the challenges of treating rare neurological diseases. Taking place online, this training will cover topics such as disease-modifying therapies, treatment of movement disorders and non-neurological disease aspects. The full programme and registration instructions are available here.
TREATM-NMD Expert LGMD masterclass 2025
From 24 to 25 April 2025
At : Montreal, Canada
From 24-25 April 2025, TREAT-NMD will be holding an expert masterclass on limb girdle muscular dystrophy (LGMD) for North American healthcare professionals. This event will deliver the latest insights on diagnosis, standards of care, and emerging therapies. Those interested in attending must apply for a spot, and successful applicants will receive funding for travel and expenses.
EpiCARE workshop on holistic care for rare and complex epilepsies
From 26 to 27 May 2025
At : Alesund, Norway
The "Beyond Seizures" working group of the ERN EpiCARE will be holding a workshop from 26-27 May on the challenges of rare and complex epilepsies beyond seizure control, promoting a more interdisciplinary approach to understanding such conditions.
TREAT-NMD SMA and DMD masterclass
From 08 to 10 July 2025
At : Prague, Czech Republic
TREAT-NMD will hold a masterclass on spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) from 8-10 July in Prague. This training is aimed at European healthcare professionals who are involved in the diagnosis and care of these conditions, and will cover topics such as diagnosis, standards of care and emerging therapies. Those who would like to attend must apply for a spot, and accepted participants will receive funding for travel and expenses.
International Summer School on Genome Stability
From 15 to 19 July 2025
At : Montpellier, France
From 15-17 July 2025, a Summer School on genome stability will be held for masters and PhD students, and postdocs, in Montpellier. The three-day training will include expert lectures, oral presentations by participants and round table discussions. The Summer School has a limit of 30 available spots, and the deadline to apply is 1 March 2025.
Grants
FDA funding opportunities for rare diseases
The FDA operates a number of programs which provide funding for rare disease research projects and organisations. A list of these programs is available here.
In addition to these programs, a list of current and open FDA rare disease funding can be found on the NIH Grants and Funding page.
ERDERA first Joint Transnational Call
The European Rare Diseases Research Alliance (ERDERA) has launched their 2025 Joint Transnational Call (JTC) for Proposals. This call, the first of its kind since ERDERA’s launch earlier this year, 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 – development and validation.”
This call is open to transnational consortia involving 4-6 principal investigator partners from at least 4 participating countries, with a maximum project length of 3 years. Proposals should address at least two of the following topics in the context of a specific rare disease or group of diseases:
- Development of novel therapies in a pre-clinical setting
- Creation and validation of predictive and pharmacodynamic biomarkers
- Replication of pre-clinical findings to enhance reliability
- Pre-clinical proof-of-concept studies for therapy readiness
This call is structured as a two-stage submission procedure, and full proposals will only be accepted from teams who have been invited to submit following the pre-proposal stage. The deadline to submit pre-proposals is 13th February 2025. More information on the eligibility criteria and submission guidelines is available on ERDERA’s website.
Job Opportunities
Scientific project manager: Functional consequences of rare diseases
Orphanet is recruiting a scientific project manager to work on the functional consequences of rare diseases. For more information, consult the job listing.
Clinical trials and orphan medicinal products intern
Orphanet is recruiting an intern currently studying science or pharmacy to work with our Expert Resources team on clinical trials in rare diseases and orphan medicinal products. For more information, consult the job listing.