The Findacure International Rare Disease Showcase
From 02 to 04 February 2022
At : Online
Events
ERN Webinar: Common challenges in uncommon conditions
On 03 February 2022
At : Online
European Cancer Meeting of the French National Cancer Institute
From 04 to 05 February 2022
At : Online
Networking event on vascular liver diseases and associated complications
From 04 to 05 February 2022
At : Online and in Vienna, Austria
EURORDIS Black Pearl Awards
On 09 February 2022
At : Online
ERN BOND 5-year anniversary conference
From 09 to 10 February 2022
At : Online
ERN GENTURIS 5-year anniversary conference
From 11 to 12 February 2022
At : Online
Global Disability Summit: 2022
From 16 to 18 February 2022
At : Online
Lessons learned from the COVID-19 pandemic: Looking to the future with The UK Rare Diseases Framework
On 23 February 2022
At : Online
International Symposium for Researchers and Clinicians on Wiskott Aldrich Syndrome- WAS2022
On 19 March 2022
At : Munich, Germany
11th Scientific Congress of the International Paediatric Transplant Association- IPTA 2022
From 27 to 30 March 2022
At : Prague, Czech Republic
20th International Vasculitis and ANCA Workshop
From 04 to 07 April 2022
At : Dublin, Ireland
International Primary Immunodeficiencies Congress – IPIC 5th Edition
From 28 to 30 April 2022
At : Vilamoura, Portugal
14th European Paediatric Neurology Society Congress- EPNS 2022
From 29 April to 03 May 2022
At : Glasgow, UK
ECTS 2022: 49th edition of the European Calcified Tissues Society Congress
From 08 to 11 May 2022
At : Helsinki, Finland
European Congress of Rheumatology - EULAR 2022
From 02 to 05 June 2022
At : Online and in Copenhagen, Denmark
World Congress on Rare Skin Diseases
From 08 to 10 June 2022
At : Paris, France
13th International Congress on Autoimmunity
From 11 to 14 June 2022
At : Athens, Greece
The European Human Genetics Conference- ESHG 2022
From 12 to 15 June 2022
At : Vienna, Austria
7th Annual TREAT-NMD International Conference
From 17 to 18 June 2022
At : Vancouver, Canada
8th Congress of the European Academy of Neurology – Europe 2022
From 26 to 29 June 2022
At : Vienna, Austria
European Conference on Rare Diseases & Orphan Products- ECRD 2022
From 28 June to 02 July 2022
At : Online
11th International Prader-Willi Syndrome Organisation Conference-IPWSO
From 07 to 11 July 2022
At : Limerick, Ireland
FENS Forum 2022
From 10 to 14 July 2022
At : Paris, France
From Paradigms to Paradoxes in Immunity and Immunopathology’ (PPII) international conference
From 07 to 09 October 2022
At : Freiburg, Germany
Third International Summit on Human Genome Editing
From 07 to 09 March 2023
At : London, United Kingdom
The Translational Science of Rare Diseases - From Rare to Care IV
From 04 to 06 May 2023
At : Tutzing, Germany
Grants
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.
Global Gene RARE Meet-Ups
Global Genes is now accepting grant applications to support a series of US-based RARE Meet-Ups to take place between April and November 2022. These events should provide critical education and much-needed in-person collaboration for rare disease patients, caregivers, and advocates at the local level to help equip participants to better serve their rare communities. Grant applications will be accepted through the Global Genes Grant Portal until Friday, February 4, 2022.
EJP RD Joint Transnational Call 2022
The European Joint Programme on Rare Diseases (EJP RD) has opened its Joint Transnational Call 2022.
The topic of this call is the "Development of new analytic tools and pathways to accelerate diagnosis and facilitate diagnostic monitoring of rare diseases". A number of national and regional funding organisations will participate in the EJP RD Joint Transnational Call 2022 and will fund multilateral research projects on rare diseases under the EJP-COFUND action.
The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with expected impact to use the results in the future for benefit of patients.
The projects shall involve a group of rare diseases or a single rare disease following the European definition, focusing on the European Community, EC associated states and Canada.
The deadline to submit the pre-proposal is 16 February 2022.
European Commission Horizon Europe Framework Programme (HORIZON)
The European Commission is funding a call on the development of new effective therapies for rare diseases with the aim of “Tackling diseases diseases and reducing disease burden.” The topic will support proposals covering several different stages in the continuum of the innovation pathway ( translational, preclinical, clinical research, validation in the clinical and/or real-world setting etc.), as relevant.
To that end, proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:
- Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
- Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and/or innovative clinical trials designs.
- Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
- Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and/or orphan medicinal products.
The deadline for this call is a two-stage model, including 1 February 2022 and 06 September 2022.
The EJPRD is offering services to help applicants for this call, notably in the fields of Data FAIRification, Clinical Studies Support Office and Mentoring.
Courses and educational initiatives
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. Three workshops are already planned in 2022 and registrations are now open.
Introduction to real world data
The European Health Data & Evidence Network (EHDEN) and the European Patients’ Forum (EPF) offer a free course to patient organisations on real world data. The course focuses on data protection and the vital role they can play in the healthcare ecosystem and health research.
The course is available upon registration through the EHDEN Academy.
ERN EpiCare e-learning modules
The ERN EpiCARE has developped new e-learning modules in collaboration with the ILAE Academy. The modules are dedicated towards professionals who wish to practice diagnosis of rare and complex epilepsies. The first online six modules are patient-centered cases.
The 8th Rare Diseases Summer School
Because of the pandemic covid-19, the 8th Rare Diseases Summer School has been postponed and will be held from 13 July to 15 July 2022 in Kartause Ittingen. The rare diseases summer school is part of the curriculum of the PhD programs. It aims to provide an environment for informal exchange between PhD students and experts in the field of rare disease research. The application procedure will open in 2022.
International Neurofibromatose Educational Resources: Masterclasses in NF
INFER (International NF Educational Resources) is a series of online educational lectures for medical professionals by leading neurofibromatosis experts.
The next lecture will be held on:
- 17th February 2022, 6pm CET:
- Emotional Challenges in NF1, NF2 and Schwannomatosis
- Chaired by Susie Henley, Thomas Pletschko, Verena Rosenmayr
ERN-Skin: Series of webinars
The ERN-Skin launches a series of scientific webinars on rare skin diseases. Each ERN-Skin thematic group organizes a one hour session, with 30 minutes presentation and 30 minutes discussion.
The next webinars will be held on:
- 22th February 2022, 1pm - 2pm CET :
- Ectodermal Dysplasias including Incontinentia Pigmenti and p63-associated disorders
- 29th March 2022, 1pm - 2pm CET :
- Autoimmune Bullous diseases
- Autoimmune Bullous diseases
Job opportunities
Scientific Project Manager "Clinical Signs in Rare Diseases"
Please send your CV and motivation letter to:
Ana Rath
jobs.orphanet@inserm.fr
Tel : +33 (0)1 56 53 81 41
Project Manager "Nomenclature and Terminology Alignments"
Please send your CV and cover letter to:
Houda Ali
Tel : +33 (0)1 56 53 81 41
jobs.orphanet@inserm.fr
Project Manager "Nomenclature and Scientific Coordination of National Hubs"
Please send your CV and cover letter to:
Houda Ali
Tel : +33 (0)1 56 53 81 41
jobs.orphanet@inserm.fr
Orphanet Nomenclature & rare diseases and X-eHealth Project Manager
Please send your CV and cover letter to jobs.orphanet@inserm.fr
Project Manager "Diagnostic Tests and Medical Laboratories"
Please send your CV and cover letter with the reference US14-2021-01 to:
Charlotte Gueydan
Tel : +33 (0)1 56 53 81 41
jobs.orphanet@inserm.fr
European Project Officer for VASCERN
The position of EU Project Officer supports the EU Project Manager in order to ensure the functioning, development and successful implementation of the VASCERN network activities (clinical care, research, teaching/training, telemedicine, patient registries, communication projects...) under the responsibility of the Coordinator and in cooperation with the network members.
Please send your CV and cover letter (in French and English) to julie.hallac@aphp.fr
Media
Euronews live debate on rare diseases
Euronews is organising a live debate on rare diseases on 15 February 2022 at 15:00 CET. The debate is entitled "Innovation in rare diseases: How can Europe be a global leader?". The panel for this debate includes Alessandra Moretti, Member of the European Parliament, Yann Le Cam, CEO of EURORDIS, and Johanna Friedl-Naderer, president of Europe, Canada and partner markets at Biogen.
The debate will address ways in which the private and public sector can cooperate in the field of rare diseases in Europe as well as the work of the European Expert Group on Orphan Drug Incentives and the devise of a policy framework that would boost the development of orphan medicinal products.
Registration is required to watch the debate.