The European Medicines Agency (EMA) is organising a joint EMA/HMA/TEHDAS Data Quality Framework for Medicines workshop as a virtual meeting on April 7th from 13.00 – 17.15 CET. This workshop seeks to engage stakeholders early in the drafting process of an EU Network data quality framework, to ensure a robust starting point, in line with their…
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As part of the EJP RD Training series, an advanced-level training webinar on the topic « Composite endpoints including patient relevant endpoints (Quality of Life) » aimed at introducing the generalized pairwise comparison ideas and concepts as well as demonstrating its benefit in small sample trials, more particularly in including patient relevant endpoints, such as quality of…
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A funded networking event “European Forum on Visceral Myopathy 2022” is being organised in Camogli, Italy. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity. The in-person event will take place over three days from April 27th – 29th. Special interest will be devoted to clinical diagnosis and patient management, genetics and onset molecular…
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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…
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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…
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A funded networking event « International Vasculitis Patient Conference » is being organised as a satellite hybrid event to the 20th International ANCA Vasculitis Workshop 2022. This event has received funding support from the EJP RD’s Networking Support Scheme (NSS) funding opportunity as part of the sixth round of funding in September 2021. The hybrid event will take place over two days from…
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The Global Alliance for Patient Access has made available a policy paper on unmet needs in Neuromyelitis Optica Spectrum Disorders (NMOSD) in Europe, published in multiple languages. A rare autoimmune condition of the central nervous system, NMOSD causes deterioration to a person’s optic nerve and spinal cord. It affects over 10,000 people in Europe but…
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Earlier this year, the Chan Zuckerberg Initiative (CZI) launched two Requests for Applications (RFA) for grant opportunities that will fund collaborative teams bringing together patient-led rare disease organizations and research teams for 4-year research projects aimed at advancing our understanding of the fundamental science of rare diseases. The Networking Expo is a two-day ideation and matchmaking workshop specifically designed…
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The Global Alliance for Global Health (GA4GH) is looking for feedback on its strategic approach via a Community Survey and eight live Town Hall meetings with members of the executive team. The Community Survey is open until May 13. The Town Hall meetings are being proposed in March and April, and are open to everyone. Every two years, GA4GH…
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Rare Conversations invites you for the inaugural episode of the new Rare Conversations event series in 2022, aimed at powering the next decade of rare disease innovation in Europe. The first episode will be focused on game-changing opportunities for the R&D community to address existing challenges in rare diseases. The aim is to understand what is the basis…
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