RDI Discussion: What is a rare disease?
On 02 February 2023
At : Online
Events
For a patient-driven evolution of the Orphan Medicinal Product Regulation: Addressing unmet needs
On 08 February 2023
At : Brussels, Belgium
Time to act: Building a pharmaceutical framework fit for rare diseases
On 08 February 2023
At : Brussels, Belgium
Third International Summit on Human Genome Editing
From 06 to 08 March 2023
At : London, United Kingdom
TREAT-NMD: virtual LGMD North American masterclass
From 09 to 10 March 2023
At : Online
As part of their series of free masterclasses, TREAT-NMD is hosting a virtual masterclass on the treatment and care of patients with LGMD aimed at healthcare professionals based in North America. The event will be held online from 9-10 March 2023, and will feature expert talks and interactive workshops. Places are limited, and registration is now open.
2023 RE(ACT) Congress & IRDiRC Conference
From 15 to 18 March 2023
At : Berlin, Germany
EJP RD – ERN Workshop: New Therapeutical Approaches for Inherited Retinal Dystrophies
From 27 to 28 March 2023
At : Valencia, Spain
In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “EJP RD – ERN Workshop: New Therapeutical Approaches for Inherited Retinal Dystrophies” is being organized by Dr Regina Rodrigo.
The in-person event will take place on 27-28 March 2023 at CIPF – Príncipe Felipe Research Center, Eduardo Primo Yúfera, 3, 46012, Valencia, Spain.
Registration is open here, and closes on December 31st.
The workshop, open for beginners and medium level researchers – junior physicians and scientists, will give an update on basic and translational research related to new therapeutic approaches for IRDs. It will bring together European experts and trainees to improve the training of young researchers on IRDs and latest therapies. Participants and speakers will involve representatives from ERN-EYE partners and non-ERN-EYE partners.
More information here
EJP-RD Training on strategies to foster solutions of undiagnosed rare disease cases
From 03 to 05 April 2023
At : Rome, Italy
The EJP-RD is holding a 3-day course entitled “Training on strategies to foster solutions of undiagnosed rare disease cases.” Through a series of expert presentations, interactive Q&A sessions, and hands-on exercises, members of the international research community and clinicians will have the opportunity to gain the instruments they need to continue working in the field of undiagnosed rare conditions.
The Istituto Superiore di Sanità is organising the course, in collaboration with a number of EJP-RD task partners.
The course will take place from 3-5 April 2023 in Rome, Italy. Registration is open now until 15 February, and selected participants will be notified by 3 March. Priority will be given to participants involved in ERNs, Undiagnosed Diseases Network International, and SOLVE-RD.
Rare Genetic Neurodevelopmental Disorders: EuroNDD 2023
From 20 to 21 April 2023
At : Amsterdam
The Translational Science of Rare Diseases - From Rare to Care IV
From 03 to 05 May 2023
At : Tutzing, Germany
EJP RD - ERN Workshop: Desmoid tumors (DTs) in patients with Familial Adenomatous Polyposis (FAP): an interdisciplinary approach
From 22 to 23 May 2023
At : Milano, Italy
In the context of EJP RD’s ERN Workshops, a workshop entitled “EJP RD – ERN Workshop: Desmoid tumors (DTs) in patients with Familial Adenomatous Polyposis (FAP): an interdisciplinary approach” is being organized by Dr Marco Vitelarro.
The in-person event will take place on 22-23 May 2023 at Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, in Milano, Italy.
Registration is open here, and closes on March 7th.
This workshop is addressed to physicians, researchers from ERN Full Members or Affiliated Partners from the junior to senior level.
More information here
Grants
Innovative Health Initiative Call 3 Open
Innovative Health Initiative's Call 3 is now open, with a number of topics open including a call around advanced therapies for rare dieases.
More information on this single stage call is available on the IHI website, linked below. The maximum financial contribution from IHI JU for the published topics is €138 million
EJP-RD Joint Transnational Call 2023
The EJP-RD is accepting submissions for their 2023 Joint Transnational Call for research projects on the topic of “natural history studies addressing unmet needs in rare diseases.” Funding is provided by a number of national and regional organisations, and will only be given to multilateral teams representing at least 4 different countries. The maximum amount of funding available varies based on the individual funding organisation.
The call has a two-stage submission procedure, with pre-proposal submissions due by 15 February 2023.
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.
AFM-Téléthon international call for proposals now open
AFM-Téléthon has opened their 2023 international calls for proposals. There are opportunities for both scientific and medical research projects on a range of topics related to neuromuscular diseases. Several different types of funding are available.
The deadline for grants and postdoctoral fellowships has now passed, but applications for PhD Fellowships will be accepted until 28 March 2023.
Courses and educational initiatives
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.
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.
