RDCA-DAP Webinar: "Teaching patients and caregivers: The role of education in research and data sharing"
On 10 August 2023
At : Online
Events
EuRRECa and EuRR-Bone Registries drop-in session
On 23 August 2023
At : Online
2023 CTX International Scientific Meeting
From 27 to 28 August 2023
At : Jerusalem, Israel
ERN RARE-LIVER: EJP RD research workshop on primary sclerosing cholangitis associated with inflammatory bowel disease
From 28 to 29 August 2023
At : Padua, Italy
Endo-ERN webinar: Klinefelter syndrome - from genomics to the clinic
On 31 August 2023
At : Online
2023 HAEi Regional Conference EMEA
From 01 to 03 September 2023
At : Munich, Germany
The Rare Fair 2023
From 07 to 09 September 2023
At : Research Triangle Park, North Carolina, USA (hybrid)
ERN meeting: Lifelong posterior hypospadias
From 08 to 09 September 2023
At : Utrecht, Netherlands
C-Path Rare and Orphan Disease Conference
From 11 to 13 September 2023
At : Washington DC, USA
Registration is now open for the 2023 Rare and Orphan Disease Conference presented by the Critical Path Institute. At this 2.5 day conference, patients, providers, researchers, clinicians, industry representatives, regulators, and scientists will learn about different aspects of drug development for rare diseases and the current activities of C-Path's different rare disease programs.
The conference will take place from 11-13 September 2023 in Washington, DC, USA. Learn more and register at the link below!
NORD National Policy & Advocacy Taskforce Meeting
On 12 September 2023
At : Online
ERN-RND webinar: Friedreich Ataxia
On 12 September 2023
At : Online
ERN-EYE GREET meeting on paediatrics
On 15 September 2023
At : Online
Global Genes Week in RARE
From 18 to 21 September 2023
At : San Diego, USA
Global Alliance for Genomics & Health 11th Plenary
From 19 to 22 September 2023
At : San Francisco, USA (hybrid)
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
ERN-EYE GREET meeting on retina
On 13 October 2023
At : Online
NORD Rare Diseases + Orphan Products Breakthrough Summit
From 15 to 17 October 2023
At : Washington DC, USA
Endo-ERN webinar: Non-classic CAH - an underdiagnosed condition
On 19 October 2023
At : Online
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
Endo-ERN webinar: Primary adrenal insufficiency - diagnosis, treatment and follow-up illustrated by cases
On 23 November 2023
At : Online
ERN-EYE GREET meeting on paediatrics
On 24 November 2023
At : Online
ERN-EYE GREET meeting on retina
On 08 December 2023
At : Online
12th European Conference on Rare Diseases & Orphan Products
From 15 to 17 May 2024
At : Brussels, Belgium (hybrid)
2nd World Congress on Rare Skin Diseases
From 12 to 14 June 2024
At : Paris, France
Courses and Educational Initiatives
RVA Rare Disease Project ECHO workshop: Rare disease patient communication and empowerment
On 24 August 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 24 August 2023, focused on patient communication and empowerment. Visit the link below for more information on how to register and get involved.
EJP RD ERN Workshop: Urogenital tissue engineering from bedside to bench and back
From 04 to 05 September 2023
At : Utrecht, Netherlands
In the framework of their ERN workshops, the EJP RD will be holding a workshop on "Urogenital tissue engineering from bedside to bench and back: What do clinicians need and what has basic science to offer." The workshop will be held from 4-5 September 2023 in Utrecht, The Netherlands, and is aimed primarily at ERN eUROGEN members. However, if spots are available, interested patients and scientists from outside the network are invited to attend. Registration is open until 15 July, 2023.
ERN EURO-NMD Neuropathies rehabilitation summer school
From 18 to 21 September 2023
At : Rome, Italy
The ERN EURO-NMD is organising a Summer School on neuropathies rehabilitation from 18-21 September, 2023 in Rome, Italy. The aim of the workshop is to educate clinicians, researchers, physiotherapists and patient advocacy groups working in rehabilitation and the NMD field on current and future modalities of rehabilitation in rare and complex peripheral neuropathies.
EJP RD International summer school on rare disease registries and FAIRification of data
From 25 to 29 September 2023
At : Rome, Italy
From 25-29 September in Rome, Italy, the Istituto Superiore di Sanità and partners are organising a training course on rare disease registries and FAIRification of data, within the context of the EJP RD. The Summer School consists of two modules, which can be registered for separately: "Rare Disease Registries," from 25-27 September, and "FAIRification of data," from 28-29 September. Three fellowships are available for participants living in an EU-13 Country or in Turkey, with an additional three available for patient representatives. Applications must be submitted by 22 May, 2023.
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.
EJP RD Leadership and Communication Skills Training for patient representatives and advocates
From 26 to 27 October 2023
At : Gdańsk, Poland
As part of the training activities proposed by the EJP RD, a course aimed at patient representatives titled "Training for patient representatives and advocates on leadership and communication skills" will be offered from 26-27 October 2023. Organised by Medical University of Gdańsk in collaboration with EURORDIS, the 2-day training and preparatory activities aims to teach participants presentation, negotiation and leadership skills through a range of activities.
The training will be free of charge for selected participants. There are also 21 fellowships available to help cover travel expenses up to 435€. The EJP RD is committed to ensuring that the training is accessible to patient representatives and advocates from all backgrounds, with those from Eastern Europe particularly encouraged to apply.
Applications for the training as well as the travel fellowship must be submitted before 26 April 2023.
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.
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.
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.
Grants
IHI upcoming opportunities
The Innovative Health Initiative has published a list of topics under consideration for inclusion in the next IHI call for proposals, to be launched in summer 2023. This is to give potential applicants additional time to start building a consortium and drafting a proposal before the official launch of the call. In particular, this includes the IHI call 4 (two-stage) topic: Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases.
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.
Jerome Lejeune Foundation autumn call for projects
The Jerome Lejeune Foundation has opened their twice-yearly call for fundamental, translational and clinical research projects that will strengthen the understanding, deepen the knowledge and/or will explore new therapeutic approaches for Down syndrome. Funding is awarded over two years in the framework of either a pilot/exploratory grant (max €50,000) or an advanced grant (max €80,000). Principal investigators of any nationality holding a long-term appointment within an academic institution are invited to apply before 13 August, 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 call “European Partnership on Rare Diseases” is part of Destination 3 “Tackling diseases and reducing disease burden.” It is a single-stage call, and the deadline is 19 September 2023 at 17:00 CEST.
The topic “Modelling and simulation to address regulatory needs in the development of orphan and paediatric medicines” is part of Destination 6 “Maintaining an innovative, sustainable and globally competitive health industry.” It is a single-stage call, and proposals must be submitted before 13 April 2023 at 17:00 CEST.
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.
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.
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.
Job Opportunities
Media
My Rett Ally: a new web app to support caregivers
The International Rett Syndrome Foundation recently released My Rett Ally, a new web app designed to simplify and enhance care coordination for families affected by Rett syndrome. It provides caregivers with a platform to record and consolidate important information, which can easily be shared with medical professionals.