TREAT-NMD: 15 years of TACT
From 31 October to 01 November 2024
At : Boston, United States
Events
ERN CRANIO 9th annual meeting
From 06 to 08 November 2024
At : Gdansk, Poland
Swiss Rare Disease Summit
On 26 November 2024
At : Bern, Switzerland
CORD & RQMO Fall 2024 Rare Disease Conference
From 26 to 27 November 2024
At : Montreal, Canada
ERICA General Assembly & ERN Research Conference
From 11 to 13 December 2024
At : Udine, Italy
8th International TREAT-NMD Conference
From 06 to 08 February 2025
At : Dubai, United Arab Emirates
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
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:
- 05/11 - ERN-RND & EURO-NMD joint webinar on Diagnostic approach to childhood-onset chorea
- 05/11 - Tuesday Lunch with RITA: Granulomas in PID and other immune dysregulated conditions
- 14/11 - ERN-RND & EURO-NMD joint webinar on DBS in Dystonia - the network perspective
- 19/11 - ERKNet/ERA webinar: Highlights from the KDIGO Clical Practice Guideline for ADPKD
- 19/11 - EURO-NMD webinar on Autonomic dysfunction in movement disorders
- 21/11 - ERN-RND & EURO-NMD joint webinar on muscle pathology of storage diseases with focus on plyglucosan and lipid storage myopathies
- 26/11 - ERN-RND & EURO-NMD joint webinar on Differential diagnosis aside from neuroimaging for non-Leukodystrophy white matter diseases
- 03/12 - ERKNet/ERA webinar on FSGS in adults
- 03/12 - Tuesday Lunch with RITA: How I treat Wiskott Aldrich Syndrome
- 11/12 - ERN GENTURIS webinar on Fertility preserving measures in hereditary cancer syndromes - the oncofertility perspective
- 07/01 - Tuesday Lunch with RITA: How I diagnose and treat monogenic causes of IBD
- 14/01 - ERKNet/ESPN webinar on Non-cystinosis Fanconi Syndrome
- 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 Advisory Committee for Therapeutics symposium
On 31 October 2024
At : Boston, United States
On 31st October 2024, TREAT-NMD's Advisory Committee for Therapeutics will be holding their first symposium discussing the development of therapies in rare neuromuscular disease. Organised as a series of interactive sessions, the symposium will give participants an opportunity to hear about the knowledge that has been gleaned over the past 15 years of the Committee's operation, including learnings from past mistakes, and valuable insights into the challenges and opportunities within the field. The symposium is designed for industry professionals involved in developing treatments for rare neuromuscular diseases, and registration is currently open.
How I treat rare lung diseases - The hummingbirds of pulmonary medicine
On 05 December 2024
At : Ingolstadt, Germany (hybrid)
On 5 December 2024, Pr Lars Henning Schmid will be organising an event titled "How I treat rare lung diseases - The hummingbirds of pulmonary medicine" (Die Kolibris der Lungenheilkunde). It is designed for German-speaking experts to exchange experiences and expand their knowledge on topics such as idiopathic pulmonary arterial hymertension, sarcoidosis, cystic fibrosis, and many more. The event is proposed in a hybrid format, with both in-person and online participation possible.
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.
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.
TANGO2 Research Foundation grant cycle
The fifth cycle of the TANGO2 Research Foundation (T2RF)'s research grant programme is now open. These grants provide essential funding for seed or pilot research projects, allowing researchers to embark on smaller-scale endeavors. The maximum funding available is $25,000 USD for single-institution projects, and $50,000 USD for multi-institution projects over a 1 year period, with an opportunity for an additional competitive one year renewal. Applications are open to clinical, basic or community researchers with a terminal degree, working in an academic medical institution or nonprofit/research institution in the US or internationally. Letters of intent must be submitted by 8 November 2024 at 17h00 EST, and the deadline for full proposals is 28 February 2025 at 17h00 EST.
Genetic Alliance UK Early career researcher award
Genetic Alliance UK has announced an award for early career researchers working with the rare disease community. The award aims to encourage and recognise the efforts of such researchers in adopting effective and inclusive approaches to patient and public involvement and engagement, and is open to any early career researcher in rare diseases in the UK. The winning researcher will receive funding to attend the annual Rare Disease Research UK conference in March 2025 in Manchester. The deadline to apply is 17 November 2024.
Foundation for Rare Diseases call for research projects on chordoma
The French Foundation for Rare Diseases, in partnership with the association AVAC, has launched a call for research projects on chordoma. This call aims to support research projects aimed at understanding the pathophysiology of chordoma, improving the diagnosis, developing better management modalities and/or therapeutic strategies, with a particular focus on:
- articulation and combination of existing treatments
- reduction of the recidivism rate
- management of pain, fatigue, side effects
This call is open to projects from all biomedical and paramedical sciences disciplines with a principal investigator belonging to a public research organisation. Selected projects will receive financial support up to €40,000 over a maximum duration of 24 months. The deadline to apply is 5 December 2024 at 17h00 CET.
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.
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: Epidemiology
Orphanet is looking to hire a Scientific project manager: Epidemiology. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Scientific project manager: Nomenclature and scientific writing
Orphanet is hiring for a Scientific project manager: Nomenclature and scientific writing. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Media
New Rare on Air and ERNs on Air podcast episodes from EURORDIS
The latest episode oof Rare on Air, EURORDIS' podcast dedicated to rare diseases, is out now. In this episode, host Julien Poulain speaks with medical student and patient advocate Zainab Alani about diagnostic delays, intersectionality, and educational gaps in rare disease medicine.
A new episode has also been released of EURORDIS' other podcast, ERNs on Air. Tune in to hear from patient representatives and ERN project managers about the value of patient journeys and how to disseminate them for maximum impact.