8th International TREAT-NMD Conference
From 06 to 08 February 2025
At : Dubai, United Arab Emirates
Events
2025 Black Pearl Awards
On 24 February 2025
At : Brussels, Belgium (hybrid)
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
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:
- 07/01 - Tuesday Lunch with RITA: How I diagnose and treat monogenic causes of IBD
- 09/01 - EURO-NMD webinar series on neuromuscular pathology: The role of biopsy in autophagic myopathies
- 14/01 - ERKNet/ESPN webinar on Non-cystinosis Fanconi Syndrome
- 16/01 - EURO-NMD webinar series on neuromuscular pathology: The role of biopsy in acquired myopathies, other than inflammatory myopathies
- 22/01 - Endo-ERN webinar on Care of adult patients with Klinefelter syndrome
- 28/01 - ERKNet webinar on Streptococcus pneumoniae-HUS
- 10/02 - Endo-ERN webinar on Update SGA, GH treatment beyond childhood
- 17/03 - Endo-ERN webinar on Long-term outcomes in CAIS
TREAT-NMD Expert myotonic dystrophy type 1 virtual masterclass
On 16 January 2025
At : Online
On 16 January 2025, the TREAT-NMD network will be holding a virtual masterclass on myotonic dystrophy type 1. Aimed at healthcare providers involved in the diagnosis and management of patients living with this disease, the masterclass will explore essential topics such as clinical presentations, psychological aspects and effective symptom management strategies.
ERN-LUNG & European Respiratory Society: Virtual school on rare lung diseases 2025
From 04 to 05 February 2025
At : Online
From 4-5 February 2025, the ERN-LUNG and the European REspiratory Society (ERS) will be organising a virtual workshop on rare lung diseases. This training is aimed at both medical professionals who may encounter such diseases in their practice, as well as patients. More information about the course content and how to register is available here.
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.
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.
Find For Rare Research Grant Initiative *EXTENDED*
Find For Rare is an independently assessed, expert-led Research Grant Initiative dedicated to improving patient care and management by providing grants to support original research projects in three lysosomal storage disorders: Fabry disease, alpha-mannosidosis, and cystinosis.
The following projects are eligible for funding:
- Projects aimed at improving understanding of the factors affecting diseases, from pathogenesis to progression
- Studies of patients' tailored clinical management
Project proposals from global regions other than the Americas are eligible for submission. Researchers are eligible to apply for grants up to €50,000 to support their project.
The call for applications has now been extended until 31 December 2024. Learn more about the eligibility criteria and application process at: findforrare.com
Second wave of EU4Health calls 2024
HaDDEA has published a second wave of calls under the EU4Health Annual Work Programme 2024, several of which may be of interest to members of the rare disease community:
- EU4H-2024-PJ-03-4 (CR-g-24-99): Personalised Cancer Medicine
- EU4H-2024-PJ-03-5 (CR-g-24-96): Supporting the establishment of new networks of expertise on cancer and cancer conditions
- EU4H-2024-PJ-03-6 (DI-g-24-76): Advancing the adoption of artificial intelligence in health
- EU4H-2024-PJ-03-7 (OA-g-24-79): Action grants for the organisation of confernece and events
The application deadline for all of the above calls is 22 January 2025 at 17h00 CET.
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.
FDA grant for clinical studies of orphan products addressing unmet needs of rare diseases
The FDA's Office of Orphan Products Development (OOPD) is currently accepting submissions for a research grant titled "Clinical studies of orphan products addressing unmet needs of rare diseases." This opportunity aims to provide funding for clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare conditions.
Letters of intent, which are optional, must be submitted by 22 September 2024. The deadline for full applications is 22 October 2024. Selected studies will receive one year of support up to a maximum of $650,000, along with future recommended support for an additional three years contingent on funding availability and recipient performance.
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.
EURO-NMD: Project Manager
EURO-NMD, the European Reference Network for the thematic grouping of rare neuromuscular diseases (NMDs) is looking for a project manager to join their team, based in Paris, France. More information on the job offer can be found on their site.