Ireland: National Workshop on Patient Rights
On 17 June 2024
At : Dublin, Ireland
Events
EU4Health Stakeholders' Conference
On 19 June 2024
At : Brussels, Belgium
10th Congress of the European Academy of Neurology
From 29 June to 02 July 2024
At : Helsinki, Finland
C-Path Global Impact Conference
From 09 to 11 September 2024
At : Washington DC, USA
EuroDysmorpho 2024
From 18 to 21 September 2024
At : Ljubljana, Slovenia
Global Genes Week in RARE
From 26 to 28 September 2024
At : Kansas City, Missouri, USA
From 26-28 September 2024, Global Genes will be holding their annual Week in RARE, featuring the RARE Health Equity Forum and RARE Advocacy Summit.
In preparation for the event, Global Genes has issued a call for special interest session proposals. Submissions are welcome for both traditional presentations and panel discussions. Learn more here.
World Orphan Drug Congress Europe 2024
From 22 to 25 October 2024
At : Barcelona, Spain
TREAT-NMD: 15 years of TACT
From 31 October to 01 November 2024
At : Boston, United States
Swiss Rare Disease Summit
On 26 November 2024
At : Bern, Switzerland
ERICA General Assembly & ERN Research Conference
From 11 to 13 December 2024
At : Udine, Italy
8th International TREAT-NMD Conference
From 06 to 08 February 2025
At : Dubai, United Arab Emirates
RE(ACT) Congress and IRDiRC Conference 2025
From 05 to 07 March 2025
At : Brussels, Belgium
2nd ERN ReCONNET International Congress on Rare and Low-Prevalence Connective Tissue Diseases
From 09 to 11 April 2025
At : Prague, Czech Republic
International Primary Immunodeficiencies Congress 2025
From 05 to 07 November 2025
At : Prague, Czech Republic
Courses and Educational Initiatives
Third EJP RD MOOC on Health Data Ethics & Regulatory Frameworks
The European Joint Programme on Rare Diseases (EJP RD) has launched their third MOOC, on the topic of “Health Data Ethics & Regulatory Frameworks in Rare Disease Research.” Developed by the French Foundation for Rare Diseases, EURORDIS, the ERN EpiCARE and the Gianni Benzi Foundation, this free online course promises valuable insights and knowledge expansion on the use of health data for research purposes in rare diseases. The course is intended to be completed over 4 weeks, with 3-5 hours of work per week. Until 28th June, expert mentors will also be actively present on the platform to answer participant questions.
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:
- 18/06 - Endo-ERN webinar on Multiple Endocrine Neoplasia Type 2: New insights from childhood to adult
- 19/06 - ERNICA research collaboration webinar: Quality of life in children with CDH
- 19/06 - ERN eEUROGEN & ERNICA joint webinar: The first core quality indicator set for Hirschprung's disease
- 19/06 - ERN ReCONNET webinar: Preconception care in patients with connective tissue diseases
- 25/06 - ERN-Skin thematic group webinar on Acquired immunological LOw prevalence and Complex AdulT diseases of thE SKIN (ALLOCATE)
- 25/06 - ERN-RND & EURO-NMD joint webinar on Autoimmune parkinsonism
- 02/07 - Tuesday lunch with RITA: How do I treat uveitis?
- 03/07 - ERN ReCONNET webinar: Explaining systemic sclerosis (SSc) to family and friends
- 17/07 - ERN ReCONNET webinar: The optimization of glucocorticoid therapy for long term treatment in connective tissue diseases
- 24/09 - ERN-RND & EURO-NMD joint webinar on Clinicala neurophysiology in dystonia
- 25/09 - Endo-ERN webinar on Turner syndrome and fertility options
- 22/10 - ERN-RND & EURO-NMD joint webinar on Neurogeriatric aspects and neurological aspects in palliative care of MSA, PSP and advanced PD
- 05/11 - ERN-RND & EURO-NMD joint webinar on Diagnostic approach to childhood-onset chorea
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.
Solve-RD industry workshop: Strategies for omics data analysis & reanalysis
On 20 June 2024
At : Online
On 20th June 2024 from 12:00-13:30 CEST, Solve-RD will be holding an online workshop presenting the strategies developed for omics data analysis and reanalysis over the course of the project. Challenges, advantages, and future developments will be discussed. The workshop is intended for representatives from industry only.
ERN RARE-LIVER 5th Autoimmune Hepatitis Research Workshop
From 27 to 28 June 2024
At : Hanover, Germany
From 27-28 June 2024, the ERN RARE-LIVER will be holding their 5th annual workshop on research in autoimmune hepatitis in Hanover, Germany. The two-day event will feature updates on ongoing clinical project, diagnostic and predictive improvements, and the understanding of AIH pathogenesis, as well as keynote lectures and a workshop on data curation and AI approaches. During registration, participants are asked to specify whether they would like to propose a new project, present an update on an ongoing project, or present preliminary research data. The workshop preceeds the 20th HepNet Symposium at Hanover Medical School, a national symposium with special sessions in English which may be of interest to participants.
EURO-NMD and TREAT-NMD 6th Neuromuscular Translational Summer School
From 09 to 12 July 2024
At : Leiden, Netherlands
From 9-12 July 2024, the ERN EURO-NMD and TREAT-NMD will be hosting their 6th annual Neuromuscular Translational Summer School. This programme provides participants with the opportunity to learn about a range of topics related to acquired and genetic neuromuscular diseases, such as the current state of the art in disease management and the drug development process. A specific emphasis will be placed on the role patients should play in each of these steps, with learnings on how to communicate research to patients and the general public.
The School will be held at Leiden University Medical Center in The Netherlands, and pre-registration is now open. Only a limited number of places are available, and registration will close once the maximum number of eligible participants is reached.
TREAT-NMD Advisory Committee for Therapeutics symposium
On 31 October 2024
At : Boston, United States
On 31st October 2024, TREAT-NMD's Advisory Committee for Therapeutics will be holding their first symposium discussing the development of therapies in rare neuromuscular disease. Organised as a series of interactive sessions, the symposium will give participants an opportunity to hear about the knowledge that has been gleaned over the past 15 years of the Committee's operation, including learnings from past mistakes, and valuable insights into the challenges and opportunities within the field. The symposium is designed for industry professionals involved in developing treatments for rare neuromuscular diseases, and registration is currently open.
Grants
Share4Rare Call4Projects
The Share4Rare project, which receives funding from the European Commission through a Horizon2020 grant, has announced the launch of the fourth 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 organisiatons and research groups whose proposed projects contribute to a deeper understanding of rare diseases.
Successful applicants will comprehensive assistance from the Share4Rare team throughout the entire research process. The application deadline is 15th July 2024 at 17:00 CEST.
Foundation for Rare Diseases Call for Proposals on new therapeutic molecules
The French Foundation for Rare Diseases has issued a call for proposals titled "Identifying new therapeutic molecules for rare diseases." It is intended to support research scientific projects that aim at identifying new molecules with potential translation to therapy to develop innovative treatments for patients living with rare diseases. Three steps of drug discovery will be considered in the call:
- Projects based on a high content/throughput screening (HCS/HTS) approach using compound libraries, towards the discovery of active molecules called "hits" with therapeutic potential.
- Projects based on a mechanistic screening focused on a reduced number of specific signaling pathways to refine the implication of targeted molecules previously identified through a larger screening.
- Projects based on the hit to lead process towards the optimisation of preidentified compounds to approach drug-like characteristics.
Proposals are welcome from research projects covering all rare diseases. Principal investigators must belong to a French research team, affiliated with academia and/or the clinical/public health sector. Early career scientists are particularly encouraged to apply as principal investigator. The maximum funding available is €40,000 per project, and the submission deadline is 3 September 2024 at 17:00 CET.
For further details on eligibility and the application process, please refer to the call text.
TREAT-NMD 2025 Conference Call for Abstracts
TREAT-NMD's 8th international conference on translational medicine in inherited neuromuscular diseases will be held from 6-8 February 2025 in Dubai, United Arab Emirates, and poster abstract submissions are currently open. Posters should align with the themes and focus areas of the conference. This year's poster theme is "Networking," and examples of welcome poster topics include:
- Collaborative projects which highlight initiatives facilitating the enhancement of healthcare for rare neuromuscular conditions through cooperation and partnership;
- Partnership working that shows how an organisation has collaborated with TREAT-NMD to benefit the global neuromuscular community;
- Utilisation of TREAT-NMD resources to advance research, clinical care, or patient advocacy efforts.
The deadline for abstract submissions is 18th October 2024, and notifications of acceptance will be sent on 8th November.
Job Opportunities
Project Manager positions with ERDERA
ERDERA is excited to announce two career opportunities for experienced professionals passionate about making a difference in rare disease research. They are looking for 2 Project Managers to join their dynamic team, starting from 01/09/2024 at Biopark, Paris (with partial remote work):
- Project Manager - Funding & Education: Oversee collaborative research funding and training initiatives within the ERDERA programme. Your role will involve coordinating activities, supporting research teams, and ensuring the successful execution of funding and training programmes.
- Project Manager - Data & Knowledge Hub: Oversee data management and expertise services. Your responsibilities will include coordination and support of activities on rare diseases data infrastructures, as well as assistance to the methodological and regulatory expertise hub.
*NEW* Scientific project manager: virtual platform for rare disease research & JARDIN
Orphanet is looking to hire a Scientific project manager: European virtual platform/data service hub for rare disease research and JARDIN joint action. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Scientific project manager: Nomenclature and terminology alignments
Orphanet is looking to hire a Scientific project manager: Nomenclature and terminology alignments. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Administrative management assistant
Orphanet is hiring for an Administrative management assistant. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.
Media
Brave RARE Ukraine: Patient stories from times of violence
The Brave RARE Ukraine project has been launched to share the stories of individuals and families living with a rare disease who have been affected by the ongoing war in Ukraine. On their website, you can view testimonies from refugees who are living with or caring for someone with a rare disease. These extremely moving stories highlight how the cruelty of war has impacted their lives, while also recognising their incredible resilience.
Not the Life We Wanted - An Inclusion Body Myositis Documentary
Recently, the documentary Not the Life We Wanted was released, highlighting the currently struggle of military veterans in the United States living with inclusion body myositis to have the disease recognised as a service-related condition. Such a recognition would improve their ability to access urgently needed medical care and assistance. You can watch the documentary here.
Complete the newsRARE International Barometer survey
Vivactis Weber, a research centre in health economics, is looking to gather insights on various aspects within the field of rare diseases for publication in the upcoming issue of the International newsRARE magazine. To this end, they are asking stakeholders in the rare disease community to complete a short survey sharing their views on three distinct themes: value-based healthcare initiatives pertaining to rare diseases; early detection and neonatal screening practices; and the policies and regulatory procedures concerning rare diseases. The survey consists of 15 questions and can be completed here.