WHA Resolution Coalition Webinar: Turning the WHA Resolution on rare diseases into action
On 28 August 2025
At : Online
Events
SMA Europe 1st International Clinical Care Symposium
From 29 to 30 August 2025
At : Milan, Italy
EURORDIS Rare Barometer survey on what helps launch webinar
On 11 September 2025
At : Online
Nordic Rare Disease Summit 2025
From 16 to 17 September 2025
At : Copenhagen, Denmark
2025 ESPE/OSCAR Science Symposium
From 18 to 19 September 2025
At : Paris, France
GA4GH 13th Plenary
From 06 to 10 October 2025
At : Uppsala, Sweden
NORD Rare Diseases & Orphan Products Breakthrough Summit
From 20 to 21 October 2025
At : Washington DC, USA
World Orphan Drug Congress Europe 2025
From 27 to 29 October 2025
At : Amsterdam, Netherlands
International Primary Immunodeficiencies Congress 2025
From 05 to 07 November 2025
At : Prague, Czech Republic
ITINERARE Symposium 2025
On 18 November 2025
At : Zurich, Switzerland
8th TREAT-NMD International Conference 2026
From 10 to 12 February 2026
At : Lisbon, Portugal
EURORDIS Black Pearl Awards 2026
On 24 February 2026
At : Brussels, Belgium (hybrid)
13th European Conference on Rare Diseases & Orphan Products
From 02 to 04 June 2026
At : Prague, Czech Republic
17th International Symposium on Sjögren's Disease
From 29 September to 02 October 2026
At : Paris, France
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:
- 02/09 - Endo-ERN, ESE & ESPE joint webinar on diagnostic and therapeutic challenges across the lifespan in CAH
- 03/09 - ERN ReCONNET webinar on microvascular involvement in antiphospholipid syndrome (APS)
- 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
- 10/09 - ERN RARE-LIVER CPMS 2.0 live case discussion on acute PVT cases - recanalization or not?
- 10/09 - ERN ReCONNET webinar on exercise in adult idiopathic inflammatory myopathies (IIMs)
- 15/09 - EuroBloodNet patient webinar on gene therapy for pyruvate kinase deficiency and congenital dyserythropoietic anemia type II
- 16/09 - TransplantChild webinar on neonatal organ donation
- 24/09 - Endo-ERN webinar on genotype-phenotype correlations in CAH
- 24/09 - ERN ReCONNET webinar on pregnancy in undifferentiated connective tissue disease (UCTD)
- 26/09 - EuroBloodNet patient webinar on introduction to sickle cell disease (SCD) and gene therapy
- 01/10 - ERN ReCONNET webinar on osteoporosis in rare and complex connective tissue diseases
- 29/10 - Endo-ERN webinar on the Clinical Practice recommendations for the diagnosis and management of XLH
- 30/10 - EuroBloodNet webinar on amyloidosis
- 04/11 - Endo-ERN, ESE & ESPE joint webinar on recent therapeutic perspectives for rare endocrine diseases
- 26/11 - EuroBloodNet patient webinar on basics of gene therapy with lentiviral vectors
2026 webinars
- 16/02 - EuroBloodNet patient webinar on genome editing: CRISPR/Cas9 and sickle cell disease - how it works and its uses
- 15/04 - EuroBloodNet patient webinar on genome editing: CRISPR/Cas9 advanced tools and sickle cell disease - using new methods
- 03/06 - EuroBloodNet patient webinar on safety of CRISPR/Cas9
- 17/07 - EuroBloodNet patient webinar on future developments and CRISPR/Cas9 for sickle cell disease
- 02/10 - EuroBloodNet patient webinar on regulatory path to the clinic
ERN RARE-LIVER workshop on quality of life in primary sclerosing cholangitis (PSC)
From 18 to 19 September 2025
At : Warsaw, Poland
Grants
ERDERA Networking Support Scheme
ERDERA has opened the first round of submissions for their new Networking Support Scheme (NSS). This continuously open call supports the organisation of transnational networking events that promote knowledge sharing, research uptake and collaborations among clinicians, researchers, and patients/patient advocacy organisations. Eligible events will have a clear rare disease or rare cancer research focus and address both aims of the call: promoting knowledge exchange and supporting greater inclusionof underrepresented countries. Funding of up to €30,000 is available per networking event.
The NSS is a permanently open call from May 2025 onwards until funds are exhausted (anticipated in April 2029). Applications will be reviewed in six-monthly collection rounds. The next collection date is 1 September 2025. More information on event eligibility and submission procedures is available on ERDERA's website.
FMR & K20 Association: Heterotopic calcifications in iPPSD
The French Foundation for Rare Diseases (Fondation Maladies Rares, FMR) and the K20 Association have launched a call for research projects on heterotopic calcifications and ossifications in inactivating PTH/PTHrP/Gsa/AMPc/PKA signaling disorders (iPPSD). This call aims to support one or more innovative, fundamental, translational or clinical research programs on this topic from any biomedical sciences discipline. The maximum financing available is €50,000 over a maximum duration of 24 months. The deadline to apply is 17 July 2025 at 17h00 CET. More information on eligibility criteria and the application procedure is available in the call text.
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.
Support for non-SSIEM international scientific meetings
The Society for the Study of Inborn Errors of Metabolism (SSIEM) is accepting applications for funding to support external international scientific meetings on inborn errors of metabolism. Applicants can request a maximum of €50,000 and must apply by 31 July 2025.
Share4Rare 2025 call for research projects
The Share4Rare project is currently accepting submissions for their 2025 call for research projects. This call seeks to support studies collecting either quantitative or qualitative data on a specific rare disease or group of diseases. Eligible projects can vary in nature, but may include:
- Patient registries
- Natural history studies
- Disease burden studies
- Creation and validation of scales and questionnaires
- Quality of life studies
- Clinical image data and patient-reported data correlation
The 2025 call will remain open until 31 August 2025. More information is available here.
FMR Call for proposals on identifying new therapeutic molecules for rare diseases
The French Foundation for Rare Diseases (Fondation Maladies Rares, FMR) is currently accepting funding applications from projects which aim to identify new therapeutic molecules for rare diseases. Eligible projects should address one or more of the following stages of the drug discovery pipeline:
- High-content/high-throughput screening (HCS/HTS)
- In vitro mechanistic screening
- Hit-to-Lead optimisation
The maximum funding available per project is €40,000, and applications must be submitted before 4 September 2025 at 17h00 CET.
FMR: Call for projects on LPAC
The French Foundation for Rare Diseases (Fondation Maladies Rares, FMR) and the ALBI France Association have launched a call for research projects on low phospholipid associated lithiasis (LPAC). The call provides for financial support of up to €30,000 over a maximum duration of 24 months. The application deadline is 11 September 2025 at 17h00 CET.
Job Opportunities
Medical writer: Rare disease encyclopedia project manager
Orphanet is recruiting a medical writer to work on our encyclopedia of rare diseases with a start date of 01/04/2026. Consult the job listing for more information, and apply by sending your CV and cover letter to jobs.orphanet@inserm.fr.
Scientific project manager - Rare disease data
Orphanet is recruiting a scientific project manager to work on rare disease data, in the context of the OD4RD2 and JARDIN Joint Action projects. Consult the job listing for more information, and apply by sending your CV and cover letter to jobs.orphanet@inserm.fr.
ERN-Skin: Project Manager
The ERN-Skin is recruiting a project manager to join their coordination team. Please find the job listing attached for more information.