ICPerMed Conference – Prelude to the Future of Medicine
From 06 to 07 October 2022
At : Paris, France
Events
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
7th International Hybrid Annual Congress of the World Muscle Society
From 12 to 16 October 2022
At : Halifax, Canada
8th International Symposium on NBIA
From 14 to 16 October 2022
At : Lausanne, Switzerland
NORD Breakthrough Summit
From 18 to 19 October 2022
At : Washington, DC, USA
The International Congress on Spinal Muscular Atrophy
From 22 to 24 October 2022
At : Barcelona, Spain
ASHG Annual Meeting 2022
From 26 to 30 October 2022
At : Los Angeles, USA
All-ePAG meeting
From 15 to 18 November 2022
At : Online
International Conference on Clinical Research Networks for Rare Diseases
From 02 to 03 December 2022
At : Paris, France
CLINICAL TRIALS IN RARE DISEASES : A VIRTUAL EVENT
On 08 December 2022
At : Online
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
EJP RD The Research Mobility Fellowships: Last call
The last call for the Research Mobility Fellowships funding opportunity has opened on October 3rd. The call aims to support PhD students, postdocs and medical doctors in training to undertake scientific visits fostering specialist research training outside their countries of residence. The exchange can be carried out either: within the same ERN (Full Members and Affiliated Partners), or between different ERNs (Full Members and Affiliated Partners), or between ERN Full Members / Affiliated Partners and non-ERN institutions.
The following categories of applicants can apply:
- PhD students with a minimum of one year of research experience at the time of submitting a proposal
- Postdocs/research fellows within five years after receiving PhD degree at the time of submitting the proposal
- Physicians with at least one year of rare disease specialist training and within five years of completion of clinical training at the time of submitting the proposal
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
EURORDIS Open Academy Schools 2023
The applications for the 2023 edition of the EURORDIS Open Academy Schools are now open.
The 2023 edition of the Open Academy Schools will take place in-person from 19-23 June in Barcelona and, following a review of our training offerings, we are excited to announce a new format for our face-to-face training.
From 2023, the in-person intensive week of 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 take place during the same week, incorporating three days of parallel sessions and two days of joint sessions focusing on patient engagement across the whole life-cycle of a medicine. Greater knowledge sharing and networking opportunities will be fostered with both sets of trainees together during the week.
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.
Registration is mandatory and open until October 24th.
The 6th edition of Duchenne Patient Academy
The 6th edition of Duchenne Patient Academy will take place on December 1 – 3 2022. This edition's theme is ‘Changing Perspectives’. The Academy provides online training for Duchenne and Becker muscular dystrophy (DMD/BMD) patient advocates. Over the course of 3 days, Duchenne Patient Academy 2022 attendees will listen to speakers and attend panel discussions moderated by experts in their respective fields. As with last year, attendees have the opportunity to become involved in the dialogue and share their experiences.
EJPRD free and online MOOC on Diagnosing Rare Diseases: New Facilitation Window
The French Foundation for Rare Diseases is delighted to announce the upcoming opening of a new facilitation window of the MOOC "Diagnosing Rare Diseases: from the Clinic to Research and back". Mentors, experts in the field, will be available online to respond to you as you progress through the MOOC.
In the context of European Joint Programme on Rare Diseases (EJP RD), the French Foundation For Rare Diseases, ERN Ithaca and ERN Genturis have co-developed this MOOC.
The goal of the course is to learn about rare genetic disease diagnosis and testing, to understand the impact of technology in diagnostic research and to gain insights into patients’ experiences of rare genetic disease.
This course is designed for individuals with a keen interest in diagnostic research and rare diseases. While primarily designed for medical students and PhD/post-doc students in biomedical sciences, it will also be of interest to Patients Advocacy Organisations’ representatives, Healthcare professionals or paramedics who want to further their knowledge of rare diseases diagnosis.
4th Neuromuscular Translational School
The Neuromuscular Translational School, organized by EURO-NMD and TREAT-NMD, focuses on therapy development from bench to bedside with a focus on neuromuscular disorders. The Translational School contains lectures about the current state of the art of acquired and genetic neuromuscular diseases, the different steps of drug development and the tools needed for this (outcome measures, biomarkers etc.). It also stresses how patients should play a role in these steps and teaches how to communicate research to patients and the general public during interactive workshop sessions.
The Translational School will take place in autumn this year from November 21-25 in Leiden University Medical Center, the Netherlands.
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. Registrations are now open for several workshops.
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
Scientific Project Manager, Medical terminology for rare diseases
Please send your CV and cover letter to:
jobs.orphanet@inserm.fr
Team Leader, Coordination of the rare disease experts international database
Please send your CV and cover letter to:
jobs.orphanet@inserm.fr
Media
Webseries "Couple and rare diseases" by the EwenLife Association
The EwenLife association offers, as part of its web series, testimonies on the theme of couples confronted to rare diseases and rare disabilities. 12 women and men share their history, their questioning, their doubts and the path they have followed until now to move forward and reinvent their life and their couple after the announcement of their child's or their spouse's rare disease, after having lived through a period of diagnostic wandering. This web series is punctuated by the interventions of two specialised psychologists and accompanied by a resource document.