ITINERARE Symposium 2023
On 30 November 2023
At : Zurich, Switzerland
Events
Conference on rare diseases and the European reference networks: How to ensure European solidarity for patients?
From 10 to 11 October 2023
At : Bilbao, Spain
RAREsummit23 - Cambridge Rare Disease Network
On 12 October 2023
At : Cambridge, UK
NORD Rare Diseases + Orphan Products Breakthrough Summit
From 15 to 17 October 2023
At : Washington DC, USA
International Symposium on Triglyceride/Neutral Lipid and Rare Diseases
From 21 to 22 October 2023
At : Kyoto, Japan
American Society of Human Genetics 2023 Annual Meeting
From 01 to 05 November 2023
At : Washington DC, USA
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
RareX 2024
From 14 to 17 February 2024
At : Johannesburg, South Africa
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)
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
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:
- 03/10 - RARE-LIVER Joint webinar on liver transplantation and vascular liver disorders (with VALDIG & EASL)
- 04/10 - GenoMed4All & EuroBloodNet webinar: Data standardisation and linkage (Standards & Federation)
- 11/10 - EuroBloodNet Topic On webinar: Suspecting inherited thrombocytopenia
- 13/10 - ERN EYE GREET meeting on retina
- 16/10 - ERN EYE webinar: Paediatric cataracts
- 19/10 - Endo-ERN webinar: Non-classic CAH - an underdiagnosed condition
- 08/11 - GenoMed4All & EuroBloodNet webinar: Data integration and analysis (Artificial Intelligence)
- 15/11 - EuroBloodNet Topic On webinar: Blood smears combined with immunofluorescence facilitate diagnosis in patients suspected for inherited platelet disorders
- 23/11 - Endo-ERN webinar: Primary adrenal insufficiency - diagnosis, treatment and follow-up illustrated by cases
- 24/11 - ERN EYE GREET meeting on paediatrics
- 08/12 - ERN EYE GREET meeting on retina
- 13/12 - Endo-ERN webinar on HNF1B disease
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 Rare Disease Project ECHO workshop: Supporting the whole family of people living with a rare disease
On 28 September 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 28 September 2023, focused on supporting family members of people living with a rare disease. Visit the link below for more information on how to register and get involved.
ESMO Preceptorship on Hereditary Cancer Genetics 2023: Paris
From 29 to 30 September 2023
At : Paris, France
From 29-30 September 2023, the European Society for Medical Oncology (ESMO) will be hosting a course on the prevention, diagnosis, treatment, and management of hereditary cancers. It is aimed at oncologists residing in Europe, but is open to all ESMO members. For successful applicants, there is no cost to apply and accommodation will be provided. A travel grant of up to 400 EUR is also available to help cover travel costs. Applications for the course will close on 27 June 2023.
EJP RD ERN workshop: CDH1 related hereditary diffuse type gastric cancer
From 12 to 13 October 2023
At : Nijmegen, Netherlands
The EJP RD is organising a workshop on "CDH1 related hereditary diffuse type gastric cancer: The shift from prophylactic total gastrectomy to optimal endoscopic surveillance" as part of their series of ERN workshops. This workshop is open to the entire ERN community, as well as PhD students, researchers, and clinicians interested in the field of hereditary diffuse type gastric cancer. Registration is open until 15 July, 2023.
EJP RD ERN workshop: Advances in regenerative medicine and tissue engineering for rare musculo-skeletal diseases
From 13 to 14 October 2023
At : Rome, Italy
From 13-14 October 2023, the EJP RD will be organising an ERN workshop on "Advances in regenerative medicine and tissue engineering for rare musculo-skeletal diseases." The workshop aims to improve the knowledge and competences on up-to-date regenerative medicine and tissue engineering technologies exploited in the design of advanced therapies for rare diseases affecting skeletal muscles and bones, and is open to the entire ERN community.
EJP RD 5th International Training Course on "Quality assurance, variant interpretation, and data management in the NGS diagnostic era"
From 18 to 20 October 2023
At : Warsaw, Poland
The 5th International Training Course on "Quality assurance, variant interpretation and data management in the NGS diagnostic era" will be organised from 18-20 October 2023 in the framework of the EJP RD. The main goal of the course is to keep researchers and clinicians up to date on the latest techniques and practices related to next-generation sequencing (NGS) and variant interpretation. It is open to participants from the international research community, including clinicians, specialists, policy makers, and patient representatives (among others) with a basic knowledge in biology or medicine. The deadline to register is June 20th, 2023.
EURORDIS Open Academy Schools 2024
Registration is now open for the 2024 edition of EURORDIS' Open Academy Schools, taking place in June 2024 in Barcelona. Taking place over one week, the Open Academy School on Medicines Research & Development (formerly EURORDIS Summer School) and the Open Academy School on Scientific Innovation & Translational Research (formerly EURORDIS Winter School) will run in parallel, providing patient advocates and PhD researchers with the chance to gain rare disease-specific, comprehensive training programs. Participants of both schools will follow a program including off-site visits to research facilities/hospitals, contact time with faculty and EURORDIS staff, and plenty of networking opportunities.
Applications for both schools closes on 27 October 2023. For more information on the specific application criteria and procedure for each school, visit the EURORDIS Open Academy website.
RVA Rare Disease Project ECHO workshop: Coordinating and connecting care for people living with a rare disease
On 26 October 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 26 October 2023, focused on care coordination and connection. Visit the link below for more information on how to register and get involved.
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.
Grants
Share4Rare Call4Projects: 3rd Open Call for Patient-Driven Research Projects
The Share4Rare project, which receives funding from the European Commission through a Horizon2020 grant, has announced the launch of the third edition of their Call4Projects. In support of Share4Rare's mission to foster the development of research projects which emphasise the perspectives of patients, applications are welcome from patient organisations and research groups whose proposed projects contribute to a deeper understanding of rare diseases. Successful applicants will receive comprehensive assistance from the Share4Rare team throughout the entire research process. The application deadline is September 30th, 2023 at 17:00 CEST.
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.
2023 EU4Health calls for action grants
The Europan Health and Digital Executive Agency (HaDEA) has published 12 calls for proposals under the 2023 EU4Health Work Programme. The topics cover a range of different health-related subjects. In particular, topic PJ-11 is for a program on orphan medical devices, in particular targeting paediatric patients. The total budget for the calls is €19,960,000. The deadline for applications is on 17 October 2023, 17h00 CET.
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.
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.
LaunchBio travel scholarships
LaunchBio is a nonprofit organisation in Silicon Valley which holds small conferences to connect rare disease biotech entrepreneurs and investors. Fondation Ipsen is offering three travel scholarships to European rare disease entrepreneurs who would like to attend a LaunchBio event to promote their company. The scholarship covers the cost of a flight, plus three nights of hotel accommodations. Interested applicants are invited to send the name of their company, the LaunchBio event they would like to attend, as well as 150-300 words explaining how they would benefit from a Fondation Ipsen Schloarship to Clea Stemitsiotis at clea.stemitsiotis@ipsen.com. The program is open until all scholarships have been used.
Job Opportunities
Scientific monitoring and content acquisition officer
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.
Document resource manager, Genes and rare disease nomenclature
Orphanet is looking to hire a Document Resource Manager, Genes and rare disease nomenclature. 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 Chief Executive Officer, Corporate and Donor Relations Assistant, and Communications Junior Manager, Projects & Programmes. More information is available here.