Joint ERICA and EJP RD Workshop “Ethics and regulatory considerations for ERN Data Access Committee members” is now reviewable!
This workshop is the fourth of the ERN data strategy workshop series. It primarily aims to train the members of the ERNs Data Access Committee on the legal and ethical aspects to consider when examining an incoming data access request.
The recording and all the presentations are now available!
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Speakers Clémence Le Cornec (ERKNet/EJPRD/ERICA – Heidelberg Universitätsklinikum) Franz Schaefer (ERKNet/EJPRD/ERICA – Heidelberg Universitätsklinikum) Faisal Ahmed (Endo-ERN – University of Glasgow) Jelena Malinina (EURORDIS) Leonardo Cervera [...]
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The Blackswan Foundation , IRDiRC and EJPRD are hosting the 7th RE(ACT) Congress and 5th IRDiRC Conference that will be held in person in Berlin, Germany from March 15th – 18th, 2023.
The joint event “RE(ACT) Congress and IRDiRC Conference 2023” aims to bring together scientific leaders and experts and young scientists from various breakthrough scientific fields to present cutting-edge research, exchange ideas, and discuss rare diseases research policies. Patients and patient organizations committed to research will also be in attendance to share their experiences and perspectives.
The RE(ACT) Congress and IRDiRC Conference 2023 represents an exciting program with [...]
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New: continuous enrollment
The third run of the MOOC (Massive Open Online Course) “Diagnosing Rare Diseases: from the Clinic to Research and back” co-developed by EJP RD, ERN Ithaca, ERN GENTURIS and the French Foundation for Rare Diseases has opened on April 18th.
Registration is free and open at this link.
We specifically encourage medical and biomedical science students to register and follow the MOOC. The topics covered include:
The diagnostic process and the types of genetic tests available for rare diseases The differences in rare genetic diseases patient pathways Technological advances for diagnostic research The [...]
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Happy to share: the birth of the Journal of Rare Diseases (JORD) published by Springer Journal of Rare Diseases is an open access, peer-reviewed journal with clinical and basic research interests in rare and genetic diseases. The journal welcomes submissions in all fields of rare diseases. In addition to original research articles, the journal publishes reviews, letters to the editor, case reports, and short communication, thereby providing a forum for discussions on cutting edge perspectives in the field of rare diseases.
Uniquely dedicated journal to rare diseases in the region. Growing interest and steadily increasing publications in the field [...]
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As part of the EJP RD Training series, an advanced-level training webinar on the topic “Does Randomization matter in RD clinical trials?” (held on 14.12.2021)
The training consists of two parts: a 1.5-hour training in less frequently applied randomization procedures and their performance on the level of evidence, followed by 1 hour of panel discussion with experts from regulatory, pharma-industrial, academic, and clinical fields.
Rare Diseases (RD) is an area where national and international multidisciplinary collaborations are valuable to overcome some of the present challenges in research. Comparative trials serve as a major basis for evaluation of new therapies in [...]
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ERICA new matchmaking tool for ERNs: inter-ERN Research Wall.
ERICA aims to promote collaborative inter- ERNs research projects. It is therefore crucial to have a centralised location to announce any new project and search for collaborators. A specific web page has been created for this purpose on the ERICA website ERN-MATCH (Please note that the current page only contains examples)
It provides basic information about the open calls for collaboration as well as contact details of the project PI. If you have any new inter- ERN project that you wish [...]
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The ERICA 2nd General Assembly took place in Bologna as a hybrid meeting.
Hosted by Istituto Ortopedico Rizzoli (IOR) and ERN BOND Coordinator Luca Sangiorgi.
The aim of the ERICA consortium, in which all 24 European Reference Networks (ERNs) take part, is to build on the strength of the individual ERNs and create a platform that integrates all ERNs research and innovation capacity.
This was a great opportunity to discuss the progress and future of the ERN related Research activities and to participate in the WP-Specific Expert Working Group sessions to exchange the success stories and to brainstorm with the [...]
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When assessing the efficacy of a treatment in any Clinical Trial, it is recommended by the International Council for Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multifaceted diseases, which is often the case in rare diseases. Therefore, the use of a combination of several clinical meaningful endpoints is preferred. Combining endpoints on a test statistics level or on the level of p-values, in general, ignores the correlation between the endpoints, while combining information on the subject level in composite endpoints does [...]
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In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Modelling & Simulation: Research Methodologies for Small Populations in Rare Diseases” aimed at facilitating discussion and exchange of knowledge on the M&S methodologies and strategies as innovative and promising enough for facing complex multifactorial or rare diseases and conditions that require highly specialised treatments and resources is being organised by Donato Bonifazi of the Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF).
The in-person event will take place over two days on July 4th – 5th at the Hotel Excelsior in Bari, Italy.
The workshop is open by [...]
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