Paris Conference for an International Childhood Cancer Data Partnership
From 07 to 08 November 2023
At : Paris, France
Events
6th International Primary Immunodeficiencies Congress (IPIC2023)
From 08 to 10 November 2023
At : Rotterdam, Netherlands
ERICA & EJP RD Joint Conference
From 20 to 21 November 2023
At : Amsterdam, Netherlands
RARE2023: The 5th International Conference on Rare Diseases
From 30 November to 01 December 2023
At : Budapest, Hungary
ITINERARE Symposium
On 30 November 2023
At : Zurich, Switzerland
RareX 2024
From 14 to 17 February 2024
At : Johannesburg, South Africa
FDA Workshop on Enhancing Adoption of Innovative Clinical Trial Approaches
From 19 to 20 March 2024
At : Hybrid
Endo-ERN Registries Symposium
From 23 to 24 April 2024
At : Milan, Italy
12th European Conference on Rare Diseases & Orphan Products
From 15 to 17 May 2024
At : Brussels, Belgium (hybrid)
European Society of Human Genetics Conference 2024
From 01 to 04 June 2024
At : Berlin, Germany
Living Rare, Living Stronger: NORD Patient and Family Forum
From 07 to 08 June 2024
At : Los Angeles, USA
2024 Rare Impact Awards
On 08 June 2024
At : Los Angeles, USA
2nd World Congress on Rare Skin Diseases
From 12 to 14 June 2024
At : Paris, France
Courses and Educational Initiatives
VASCERN Summer School 2024
From 05 February to 28 September 2024
At : Paris, France and online
Applications are now open for the inaugural edition of the ERN VASCERN’s European Summer School. This program aims to train young doctors on the subject of rare vascular diseases, as well as to perpetuate and expand the network’s objectives to the new generation of healthcare professionals in Europe. It will be delivered in two modules: an online component, from 5th February to 17th May, 2024; and an in-person course, which will be held from 23rd-28th September, 2024 in Paris, France. Applications are open to medical students (undergraduate, masters or PhD level) and interns at European universities, and must be submitted before 19th November, 2023.
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:
- 07/11 - ERN-ITHACA webinar: Guidelines from Start to Finish: An overview of the methodology
- 08/11 - GenoMed4All & EuroBloodNet webinar: Data integration and analysis (Artificial Intelligence)
- 09/11 - ERN-RND & EuroNMD joint webinar: Pathology of peripheral nerves
- 09/11 - ERN GENTURIS webinar on Lynch syndrome - the gastroenterologist's perspective
- 14/11 - ERN-RND & EuroNMD joint webinar: Diagnostic workup and prognostic implication for sleep disorders in atypical parkinsonisms
- 15/11 - EuroBloodNet Topic On webinar: Blood smears combined with immunofluorescence facilitate diagnosis in patients suspected for inherited platelet disorders
- 22/11 - ERN GENTURIS webinar: CTNNA1 germline variants: genotype-phenotype associations
- 23/11 - Endo-ERN webinar: Primary adrenal insufficiency - diagnosis, treatment and follow-up illustrated by cases
- 24/11 - ERN EYE GREET meeting on paediatrics
- 30/11 - ERN-RND & EuroNMD joint webinar: Myopathology in Protein Aggregate Myopathies and vacuolar myopathies
- 05/12 - ERN-RND & EuroNMD joint webinar: Diagnostic workup for genito-urinary dysfunctions in atypical parkinsonism
- 08/12 - ERN EYE GREET meeting on retina
- 13/12 - Endo-ERN webinar on HNF1B disease
- 10/01 - Endo-ERN webinar on Gonadal dysfunction in males with CAH
- 18/01 - EuroNMD webinar on rhabdomyolysis
- 25/01 - ERN-RND & EuroNMD joint webinar on distal myopathies
EJP RD Funded ERN Training Workshops
The EJP-RD is holding workshops aiming at training ERN researchers and clinicians in relevant innovative themes with a cross-ERN added value. These 2-days ERN workshops are open to interested persons (clinicians/scientists) affiliated to ERNs or Affiliated Partner Institutions.
Training themes may include innovative research methodologies, diagnostic research methodologies, interdisciplinary treatment approaches, such as gene therapy and transplantation, etc. Topics have to be proposed by the ERNs or by investigators belonging to EJP RD beneficiary institutions. Registrations are now open for several workshops.
Recordati Foundation 2023 live courses programme
Throughout the year, the Recordati Foundation is organising a series of courses to help advance knowledge in rare diseases. The programme contains the following sessions:
- Genetic therapies: 22-24 June in Heidelberg, Germany
- Non-adenomatous pituitary pathology: 26-28 October in Basel, Switzerland
- Early diagnosis and early treatment of inherited metabolic diseases: 23-25 November in Tokyo, Japan
For more information about the content of the courses and details about registration, please visit the Foundation's website.
RVA Project ECHO workshop: Innovative approaches to rare disease prevention and care
On 23 November 2023
At : Online
Rare Disease Project ECHO is a component of the RArEST project, seeking to strengthen clinical learning practice for rare diseases. Regular videoconferencing sessions connect community providers with a multidisciplinary team of experts, who are able to equip communities with timely, necessary knowledge.
The next session will take place on 23 November 2023, focused on Whole of Life Care. Visit the link below for more information on how to register and get involved.
ERN-EYE workshop on genomics and personalised medicine
From 07 to 08 December 2023
At : Strasbourg, France
The ERN-EYE will be holding their next workshop from 7-8 December 2023 in Strasbourg, France on "Rare eye diseases: challenges and dilemmas in the era of genomics and personalised medicine."
ERN-RND and EURO-NMD joint webinars 2023
The European Reference Network for Rare Neurological Diseases (ERN-RND) organises joint free educational webinars on rare neurological and movement disorders with EAN. These 1-hour webinars take place throughout the year and are presented by international experts. They discuss various aspects from more general clinical features, examination, disease diagnosis, medical interventions, and disease management to more specific ones as use of scales or imaging. Adult and paediatric neurology are both covered.
Further information about ERN-RND webinars in collaboration with EURO-NMD and the European Academy of Neurology (EAN) is available here.
TREAT-NMD Expert BMD Masterclass 2023
From 07 to 08 December 2023
At : Barcelona, Spain
The TREAT-NMD network will be holding an expert masterclass on Becker muscular dystrophy from 7-8 December 2023 in Barcelona, Spain. This in-person event is aimed at healthcare professionals and researchers who are or will become involved in the diagnosis, care, and treatment of patients with BMD in Europe.
EJP RD workshop: Managing RD biological sample data in Biobanks
From 13 to 14 December 2023
At : Milan, Italy
The EJP-RD is offering a training workshop from 13-14 December 2023 on how to manage rare disease biological sample data in biobanks. Aimed at biomedical researchers, medical professionals, and biobank managers who want to develop the capacity on data mangement of biobanks, the workshop will explore the high value of biological sample repositories for rare disease research. Registration is open to healthcare professionals, researchers, clinicians, biobank managers, database managers, and patient advocates. Travel fellowships are available. Prospective participatns are asked to apply by 23rd October 2023.
ERN RITA: Immune dysregulation workshop by the SJD Barcelona Children's Hospital
On 18 December 2023
At : Barcelona, Spain (hybrid)
On 18 December 2023, a hybrid workshop on immune dysregulation is being organised by members of the ERN RITA in collaboration with the University Hospital Sant Joan de Déu in Barcelona. Participation is free, and all interested parties are invited to attend.
Grants
Multiple grant opportunities from NORD
The National Organization for Rare Disorders has announced the following new funding opportunities:
- Up to $35,000 for scientific and/or clinical research studies related to Arteriovenous Malformation (AVM). The submission deadline is 16 October 2023.
- Up to $40,000 for scientific and/or clinical research studies related to Levy-Yeboa syndrome (LYS). The submission deadline is 16 October 2023.
- $30,000 for scientific and/or clinical research studies related to megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS). The submission deadline is 16 October 2023.
More information about the specific calls is available on NORD's website.
IQSEC2 Research and Advocacy Foundation Research Grant Program 2023
The IQSEC2 Research and Advocacy Foundation is offering a 1-year grant to support research related to IQSEC2-related disorder. The Foundation will award either one grant of $60,000 or two grants of $30,000. All individuals holding a faculty-level position or a senior position at a non-profit institution or foundation are eligible to apply, including researchers with no prior experience with IQSEC2. Applications must be submitted by 4 November 2023.
Canadian Institutes of Health Research: Funding opportunities for rare disease projects
The Canadian Institutes of Health Research has announced several new funding opportunities for rare disease projects. The first team grant, "Improving Diagnosis for Rare Disease Patients," aims to support research which will determine the best pathway to diagnose RDs by moving genomic testing towards the front end of the care pathway. The maximum amount per grant is $458,333 per year for up to 3 years. The submission deadline is 7 November 2023.
The second opportunity, "Improving Health and Administrative Data and Monitoring for Rare Diseases," aims to support implementation research to determine the prevalence, direct cost, and burden of rare diseases in the Canadian health care systems. The maximum amount per grant is $400,000 per year over four years. The submission deadline is 7 November 2023.
Horizon Europe Health Calls 2023
The European Commission's Horizon Europe Health Calls 2023 are now open, with several funding calls specific to rare diseases:
The topic "Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases" is part of the Innovative Health Initiative JU Call 4. It is a two-stage call, with an initial submission deadline of 8 November 2023 at 17:00 CET.
Medics for Rare Diseases Student Voice Prize 2023
The 2023 edition of Medics for Rare Diseases' Student Voice Prize is now open for applications. This international essay competition offers applicants the chance to have their work published in the Orphanet Journal of Rare Diseases, to present at M4RD's annual conference in London, and to receive cash prizes. It is open to all students of medicine, nursing, and biological sciences. The submission deadline is 15th November 2023 at 12pm GMT. More information on the submission and eligibility criteria is available here.
FDA funding opportunity for rare disease research
The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) has announced a grant to support efficient and innovative natural history studies that advance medical product development in rare diseases with unmet needs. The funding opportunity announcement is entitled “Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) Clinical Trials Not Required”. The purpose of this grant is to address critical knowledge gaps and facilitate rare disease product development. The receipt dates for this funding opportunity are 15 February 2022 and 13 February 2024.
Another funding opportunity from the OOPD is available for fiscal years 2023-2025 to support 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 diseases or conditions. The grant is titled "Clinical studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)." The receipt dates for this opportunity are 24 October 2023 and 22 October 2024.
Job Opportunities
Rare disease research database manager
Orphanet is looking to hire a Rare Disease Research Database Manager. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Scientific monitoring and content acquisition manager
Orphanet is looking to hire a Scientific monitoring and content acquisition officer. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Job opportunities at EURORDIS
EURORDIS is looking to fill multiple positions, including Compliance & Budget Manager, Chief Executive Officer, Corporate and Donor Relations Assistant, and Communications Junior Manager, Projects & Programmes. More information is available here.