In the context of EJP RD’s ERN Workshops, a face-to-face workshop entitled “Functional exploration of genetic variants in cardiac diseases” aimed at presentation of functional explorations of variants of interest identified using Drosophila, C. elegans, Zebrafish and iPSc models to understand the molecular bases of genetic diseases, in particular heart and muscle diseases, is being organised by Philippe Chevalier of the Hospices Civils de Lyon.
The in-person event will take place over two days on June 14th – 15th at the Hospices Civils de Lyon in Lyon, France.
The workshop is open by prior registration and selection to cardiologists, [...]
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Drug development programmes in rare diseases have many challenges, some of which differ from those facing researchers working on common diseases, like the lack of clinical research experts and the scarcity of patients. Moreover, academic sponsors and investigators end up getting involved not only in the scientific aspects of the research but also having to navigate the operational coordination and management themselves. The Rare Diseases Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.
The toolbox aims to collect the accumulated knowledge, experience, and [...]
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This workshop is the third of the ERN data strategy workshop series. It is dedicated to the various stakeholders expected to interact with the EJP RD Virtual Platform.
In this workshop, the feedback received from the ERN registries on the level of discovery that should be authorised for either anonymous or authenticated users of the Virtual Platform will be discussed first. The practical use of the VP will be presented in a Demo session. Subsequently, a hands-on session will allow participants to test the Virtual Platform and provide their feedback. Researchers as well as stakeholders from industry, the member states [...]
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We are glad to start the new ‘ERICA year’ with the Kick-off meeting of WP3 Patient Centred Expert Working Group (PCEWG) that will be held in two consecutive sessions on Tuesday 8th and Thursday 10th March 2022 from 2 to 5 pm (CET).
Session I will be dedicated to present the work done so far for the identification and codification of instruments for the assessment of Patient Reported Outcomes (PRO) in rare diseases (RD). Session I will give the frame for an open discussion in Session II to better understand and define the RDs community needs in terms of building [...]
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The pilot project to support the repurposing of medicines was launched at the end of October 2021 by EMA and the Heads of Medicines Agencies (HMA)
To support not-for-profit organisations and academia ahead of the upcoming deadline for submission (28th February 2022), EMA is hosting a ‘walk-in’ clinic webinar on 17 February 17h-18h30. Representatives of the Repurposing Observatory Group leading the pilot will also participate to the webinar.
EMA together with representatives of the Repurposing Observatory Group (RepOG) is organising this open event to:
provide a brief reminder of the Repurposing pilot provide information on how EMA real-world evidence [...]
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A hands-on session will be held for you (as Virtual Platform (VP) end-users) to test the first version of the VP, provide feedback and address any further open questions.
This workshop is the second of the ERN data strategy workshop series. In this workshop, different approaches to making data and cases safely findable via the EJP RD Virtual Platform (VP) will be described, along with a short technical overview of how this is achieved.
This includes an elaboration of the difference between Resource Discovery (incorporating completely safe querying of very limited/obfuscated data fields) and far deeper Record Interrogation queries by [...]
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The way clinical trials are conducted in the EU will change when the Clinical Trials Regulation (Regulation (EU) No 536/2014) becomes applicable and the Clinical Trials Information System (CTIS) goes live on 31 January 2022.
The EMA is organising a webinar to remind SMEs and academic sponsors of clinical trials of the main changes brought by the Regulation and its impact on their trials-related activities.
The aim of the training is to give an overview of the Regulation and introduce the new process for submitting clinical trials information in the EU/EEA. In addition, it covers the functionalities of [...]
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Happy to announce that the ERICA Advisory Board is complete!
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In the context of EJP RD’s ERN Workshops, a face-to-face workshop on “Endocrine cancer: A challenge in adults and children” aimed at giving an update on the molecular background and clinical management of rare endocrine malignancies is being organised by Prof. Barbara Jarzab of the M. Sklodowska-Curie National Research Institute.
The in-person event will take place on May 4th, 2022 at the M. Sklodowska-Curie National Research Institute in Gliwice, Poland.
Registration closes on January 28th, 2022
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