8th Congress of the European Academy of Neurology – Europe 2022
From 26 to 29 June 2022
At : Vienna, Austria
Events
European Conference on Rare Diseases & Orphan Products- ECRD 2022
From 28 June to 02 July 2022
At : Online
Tour de France of the digital health acceleration strategy
On 30 June 2022
At : Strasbourg, France
TAKING CPSS TO THE NEXT LEVEL – 2ND MEETING OF EXPERTS
From 02 to 03 July 2022
At : Geneva, Switzerland and online
International Congress on Neuromuscular Diseases- ICNMD 2022
From 06 to 10 July 2022
At : Brussels, Belgium
The Cambridge Rare Disease Showcase
On 07 July 2022
At : Cambridge, UK
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
7th International Myology Congress
From 13 to 16 September 2022
At : Nice, France
3rd International Workshop on Klinefelter Syndrome, Trisomy X, and XYY
From 13 to 15 September 2022
At : Leiden, the Netherlands
International Scientific Symposium on the Ehlers-Danlos Syndromes and Hypermobility Spectrum Disorders
From 15 to 19 September 2022
At : Rome, Italy and Online
From Paradigms to Paradoxes in Immunity and Immunopathology’ (PPII) international conference
From 07 to 09 October 2022
At : Freiburg, Germany
Innovation Bootcamp in Rare Diseases 2022 (IBRD2022)
On 12 October 2022
At : Brussels, Belgium
The International Congress on Spinal Muscular Atrophy
From 22 to 24 October 2022
At : Barcelona, Spain
Third International Summit on Human Genome Editing
From 07 to 09 March 2023
At : London, United Kingdom
2023 RE(ACT) Congress & IRDiRC Conference
From 16 to 19 March 2023
At : Berlin, Germany
The Translational Science of Rare Diseases - From Rare to Care IV
From 04 to 06 May 2023
At : Tutzing, Germany
Grants
Friederichs’s Ataxia research grants: 2022 programme
In order to stimulate research and studies on Friedreich's Ataxia, the French Friedreich's Ataxia Association (A.F.A.F.) will be offering in 2022 grants up to 30.000 €/year/project, for a maximum of two years. The grants should be awarded in November 2022. The fields considered will include the basic aspects of the disease (genetics, biochemistry, neurobiology), clinical studies or more fundamental research in animal models. All the applications must clearly indicate the expected consequences on the treatment of the disease. Projects including therapeutic studies or research aiming at identifying new therapeutic strategies for Friedreich’s ataxia will be particularly encouraged. Projects with a clear impact on the patients’ quality of life will equally be appreciated (e.g., physiotherapy, speech therapy or focusing on vision and hearing problems...) as well as grant applications that foster international collaboration.
Grant application forms are available from AFAF:
Madeleine SCHMEDER
00336 37 47 67 78
Application deadline: August 26th 2022
NORD's Grant Funding Available for Rare Disease Research
NORD has announced three new requests for proposal for grant funding related to the following rare diseases: Autoimmune Polyglandular Syndrome Type 1 (APS-1), Levy-Yeboa Syndrome (LYS), and Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS).
- With funding from the APS Type 1 Foundation, NORD is accepting applications for one grant of $50,000 for scientific and/or clinical research studies related to APS-1. Deadline for letters of intent is Tuesday, June 21.
- With fundraising by the Maxwell Family, NORD is accepting applications for one grant up to $40,000 for scientific and/or clinical research studies related to LYS. Deadline for letters of intent is Tuesday, June 21.
- With funding from the MMIHS Foundation, NORD is accepting applications for one grant of $30,000 for scientific and/or clinical research studies related to MMIHS. Deadline for letters of intent is Tuesday, June 21.
Grants will be awarded to qualified researchers to initiate small scientific research studies or clinical trials, the results of which could be used to obtain funding from the National Institutes for Health (NIH), US Food and Drug Administration (FDA), or other funding agencies, or to attract a corporate sponsor.
EIC funding call for RNA-based therapies and diagnostics for rare genetic diseases
The European Commission (EC) has adopted the 2022 work programme of the European Innovation Council (EIC), opening funding opportunities worth over €1.7 billion in 2022 for breakthrough innovators to scale up and create new markets. This includes €60.5 million to tackle three Transition Challenges, notably one on the development of RNA-based therapies and diagnostics for complex or rare genetic diseases.
The EIC Transition 2022 (HORIZON-EIC-2022-TRANSITION-01) call open on 1st March is intended to meet the following specific objectives:
- Advance, beyond the state-of-the-art, RNA delivery methods, including robust mRNA formulations, that would enable effective and safe delivery of mRNA into the cells;
- Design, develop and preclinical validate of novel miRNAs (miRNA lncRNA, tRNA or siRNA-based) therapies for complex or rare genetic diseases;
- Develop and validate novel RNA-based diagnostics and RNA-based predictive biomarkers that would allow for early and more accurate diagnosis and for favourable or non- post-treatment prognosis, respectively.
The starting point in the project should be a preliminary technology or protocol of an RNA-based therapy for complex or rare genetic diseases with unmet medical needs that demonstrates, in a lab or preclinical context, the essential features that underpin the disruptive nature of the innovation. The endpoint in the project should be a completely functional version of the technology suitable for clinical validation, supported by a sound and implementable commercialisation/exploitation strategy.
The funding call has the following deadlines: May 4th and September 28th.
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.
Courses and educational initiatives
Joint EJP RD ERICA Workshop: “Ethics and regulatory considerations for ERN Data Access Committee members”
A workshop entitled “Ethics and regulatory considerations for ERN Data Access Committee members” and jointly organised by ERICA and EJP RD will take place online on 30 June 2022. It primarily aims to train the members of the ERNs Data Access Committee members on the legal and ethical aspects to consider when examining an incoming data access request. All other interested stakeholders can also join the workshop.
In this workshop, the type of data sharing that the ERNs envision with the different stakeholders will be presented. The role of the data access policy in regulating the access to the data will be explained and the structure of the data access request and of the data access request feedback form will be then presented, together with some practical aspects of the DAC work.
EURORDIS Open Academy
The EURORDIS Open Academy provides courses to empower patient advocates with knowledge and skills to take part in patient engagement roles with all stakeholders and to advocate for rare diseases on a European and national level.
Trainings are delivered in English and comprise-learning modules, pre-training webinars and face-to-face/online intensive days.
RareLaunch programme
NORD’s RareLaunch programme provides an accessible educational platform that allows an individual to explore the steps to start a nonprofit and/or to engage in starting or expanding research efforts as a nonprofit leader. Twi courses are available, Forming a Foundation and Research Ready. Forming a Foundation provides education and training for interested patients and caregivers looking to form rare-disease-focused nonprofits. Research Ready supports the efforts of patient organizations to effectively prepare for scientific research and the creation of rare disease registries. Both courses emphasize capacity building and expanding confidence at a pivotal stage in a rare disease community’s growth and development.
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.
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.
Job opportunities
Administrative Assistant
Please send your CV and cover letter to:
jobs.orphanet@inserm.fr
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
Scientific Project Manager Medical terminology for rare diseases
Please send your CV and cover letter to:
jobs.orphanet@inserm.fr