ERNs needs on data analysis (questionnaire)

EJP RD Bring Your Own Omics Data workshop (BYOOD) workshop (November 2022)

ERNs needs on data analysis (questionnaire)

The Bring Your Own Omics Data workshop (BYOOD) workshop (November 2022) is aiming to help ERNs to optimise and integrate their omics data, initiate network analysis, and continue collaborations with the data analysis team.

If you are part of the ERN, please help the EJP RD workshop organising team to further understand what your ERNs needs on data analysis are and complete this questionnaire by the 07th of July 2022 through the following link (takes only few minutes!):

https://forms.office.com/r/62EtZpwfV7

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Launch of the Innovation Management Toolbox (IMT)

EJP RD is launching on June 30th, 2022 the Innovation Management Toolbox (IMT), a free-to-use and curated reference library of resources in rare disease translational medicine that will provide investigators with self-help resources specific to their needs. The database will be maintained actively!
Learn about the IMT: https://www.ejprarediseases.org/innovation-management-toolbox/ Access the IMT: https://imt.ejprarediseases.org/

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The webinar on “Composite endpoints including patient relevant endpoints (Quality of Life)”video now available

The webinar on “Composite endpoints including patient relevant endpoints (Quality of Life)” is now available here

In this webinar you will learn about:

The procedures to combine multiple endpoints and its limitations The properties and the flexibility of the class of generalized pairwise comparison tests The potential advantages and disadvantages of designing a clinical trial in rare disease with generalized pairwise comparisons primary analysis

You can watch also webinar on Randomization procedures in Rare Disease Clinical Trials here.

All the available ERN Research related webinars: https://erica-rd.eu/events/webinars/

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“Ethics and regulatory considerations for ERN Data Access Committee members”

We are happy to invite you to take part in a workshop addressing “Ethics and regulatory considerations for ERN Data Access Committee members”.

This workshop is jointly organised by ERICA and EJP RD and will take place online on 30th June from 10 am to 12:30 pm.

It primarily aims to train the members of the ERNs Data Access Committee members on the legal and ethical aspects to consider when examining an incoming data access request. All other interested stakeholders are welcome to join.

See the detailed agenda (, 213 KB).

Please [...]

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RE(ACT) Congress & IRDiRC Conference-SAVE THE DATE 15-18 March 2023

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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MOOC on Diagnosing Rare Diseases: from the Clinic to Research and back

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 role of collaborative [...]

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Journal of Rare Diseases (JORD)

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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Training Webinar: Does Randomization matter in RD clinical trials?

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 Collaborative inter-ERN Research Wall

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 Research Wall (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 [...]

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