EuroDysmorpho 2024
From 18 to 21 September 2024
At : Ljubljana, Slovenia
Events
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
ERN CRANIO 9th annual meeting
From 06 to 08 November 2024
At : Gdansk, Poland
Swiss Rare Disease Summit
On 26 November 2024
At : Bern, Switzerland
CORD & RQMO Fall 2024 Rare Disease Conference
From 26 to 27 November 2024
At : Montreal, Canada
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
EUCAPA HTA Regualtion trainings
European Capacity Building for Patients (EUCAPA) has a number of online trainings available for patients who would like to learn more about the HTA regulation and its implications for patient engagement.
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:
- 24/09 - ERN-RND & EURO-NMD joint webinar on Clinicala neurophysiology in dystonia
- 25/09 - ERN GUARD-Heart webinar on Cardiac rehabilitation in congenital heart disease
- 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.
Recordati Foundation course on adrenal failure
From 17 to 19 October 2024
At : Stockholm, Sweden
The Recordati Rare Diseases Foundation is organising a course for adult and paediatric endocrinologists titled "Adrenal failure: update on causes, diagnosis, treatment, crisis and life expectancy." It will focus on defining and exploring all types of adrenal failure, with a focus on topics such as diagnostic challenges, autoimmune mechanisms, and treatment modalities. The training will be held in-person in Stockholm, Sweden from 17-19 October 2024. More information about registration, fees, and selection criteria is available here.
EURORDIS 6th Mental health & wellbeing webinar
On 23 October 2024
At : Online
On 23 October 2024, EURORDIS will be hosting their latest mental health and wellbeing webinar from 14h-15h30 CET on the topic "How to deliver a diagnosis." During the webinar, experts from ERNs will present real cases and best practices for communicating a rare disease diagnosis in order to ensure that patients, as well as their families and caregivers, receive adequate psychosocial support.
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.
How I treat rare lung diseases - The hummingbirds of pulmonary medicine
On 05 December 2024
At : Ingolstadt, Germany (hybrid)
On 5 December 2024, Pr Lars Henning Schmid will be organising an event titled "How I treat rare lung diseases - The hummingbirds of pulmonary medicine" (Die Kolibris der Lungenheilkunde). It is designed for German-speaking experts to exchange experiences and expand their knowledge on topics such as idiopathic pulmonary arterial hymertension, sarcoidosis, cystic fibrosis, and many more. The event is proposed in a hybrid format, with both in-person and online participation possible.
Grants
FDA funding opportunities for rare diseases
The FDA operates a number of programs which provide funding for rare disease research projects and organisations. A list of these programs is available here.
In addition to these programs, a list of current and open FDA rare disease funding can be found on the NIH Grants and Funding page.
Galactosemia Foundation Research Grant Program
The Galactosemia Foundation is now accepting submissions for the 2024-2026 cycle of their Research Grant Program. Submissions are welcome for research projects, clinical trials, educational projects, and collaborative projects or meetings hoping to improve the lives of those living with galactosemia. The foundation is particularly interested in submissions which address the following topics:
- Premature ovarian insufficiency/failure
- Motor/neurological deficits
- Cognition
- Advancements in diet-related issues
- Bone density and bone health in classic galactosemia (male/female) including advances in treatments and relationship with hormones and/or metabolism
Funds are allocated on an annual basis, and the usual funded project duration is one year. Grants typically range from $10,000 to $50,000. Project selection is conducted in a two-stage process, and the submission deadline is 30 September 2024.
EU4Health call for tenders: Study on the provision of care for adolescent and young adult cancer patients in the EU
The Health and Digital Executive Agency (HaDEA) has issued an EU4Health call for tenders for a study on the provision of care for adolescent and young adult (AYA) cancer patients in the EU. This study aims to map the provision of healthcare to AYA cancer patients and survivors in all EU countries, Norway, and Iceland, and to document and analyse the major barriers and enabling factors for high quality care. The maximum funding ceiling is €1,000,000. The submission deadline is 9 October 2024 at 16h CEST.
Find For Rare Research Grant Initiative
Find For Rare is an independently assessed, expert-led Research Grant Initiative dedicated to improving patient care and management by providing grants to support original research projects in three lysosomal storage disorders: Fabry disease, alpha-mannosidosis, and cystinosis.
The following projects are eligible for funding:
- Projects aimed at improving understanding of the factors affecting diseases, from pathogenesis to progression
- Studies of patients' tailored clinical management
Project proposals from global regions other than the Americas are eligible for submission. Researchers are eligible to apply for grants up to €50,000 to support their project.
The call for applications is open until 31 October 2024. Learn more about the eligibility criteria and application process at: findforrare.com
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.
FDA grant for clinical studies of orphan products addressing unmet needs of rare diseases
The FDA's Office of Orphan Products Development (OOPD) is currently accepting submissions for a research grant titled "Clinical studies of orphan products addressing unmet needs of rare diseases." This opportunity aims to provide funding for clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare conditions.
Letters of intent, which are optional, must be submitted by 22 September 2024. The deadline for full applications is 22 October 2024. Selected studies will receive one year of support up to a maximum of $650,000, along with future recommended support for an additional three years contingent on funding availability and recipient performance.
Job Opportunities
Scientific project manager: Epidemiology
Orphanet is looking to hire a Scientific project manager: Epidemiology. 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 scientific writing
Orphanet is hiring for a Scientific project manager: Nomenclature and scientific writing. The job listing is available here.
If interested, please send your CV and cover letter to jobs.orphanet@inserm.fr.