The AKU Society, a UK-based charity, has launched a crowdfunding campaign to raise funds for « Leave No Patient Behind – A Global Black Bone Disease Registry » in order to gather information about alkaptonuria (AKU) patients around the world in one single place. Alkaptonuria, also known as AKU or Black Bone Disease, is an extremely rare genetic condition,…
The 1st International Conference on Rare Diseases and Paediatric Research is being organized by 95, Rare Alliance Greece together with Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) and « Athena » Research Center in Information, Communication and Knowledge Technologies. The Conference is aimed at opening the discussion with all relevant stakeholders on the challenging topic of research…
EJP RD is co-organising the « Rare Conversations – European Rare Disease Ecosystem: A Collaborative Path Forward » conference in cooperation with Alexion, EURORDIS, EUCOPE, and EuropaBio. The conference is targeted towards representatives of the different communities active in rare diseases: patients, researchers, clinicians, regulators, investors, payers, and industry. The fully online conference will take place on November 9, 2021 from 14.00 – 18.00 CET.…
The Professional Society for Health Economics and Outcomes Research (ISPOR) is organising a webinar entitled “Health Preference Research in the Context of Gene Therapy in Rare Diseases” that will be limited to 1000 registered participants on a first-come, first-served basis. The webinar will take place on September 27, 2021 from 16.00 – 17.00 CET. This webinar will present the value…
The National Organization for Rare Diseases (NORD) is organising its annual NORD Summit 2021, one of the largest multi-stakeholder events in rare disease, bringing together rare disease community stakeholders, including rare disease experts and leaders from patient advocacy groups, government, industry, and academia to discuss the most current and critical topics related to rare diseases…
The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) is organising a multi-stakeholder round table discussion on the theme of the future of rare diseases in Europe: Revision of the European Orphan & Paediatric Regulation and impact for the Nordic Rare Disease Community in the context of review of the European legislation on paediatric medicines (EC 1901/2006)…
The United Nations Political Forum on Sustainable Development (HLPF) is co-organising a High-Level Side Event titled « On the road towards COVID-19 recovery & delivery of the Sustainable Development Goals – Addressing the challenges of persons living with a rare disease as a human rights, sustainable development and equity priority » along with the NGO Committee for…
As part of the training activities proposed by the EJP RD, the International Summer School on Rare Disease Registries and FAIRification of Data is a 5-day online training programme organised by Istituto Superiore di Sanità (ISS) in close collaboration with EJP-RD task partners, aimed at the international research community, clinicians, medical specialists, registry curators, database…
EJP RD is organising an integrative research and innovation strategy meeting on the theme of alignment of national rare disease strategies with the EJP RD targeted towards policy makers and persons involved in national, EU, and international rare diseases strategies. The fully online strategy meeting will take place on 8th July 2021 from 09:30 – 17:00 CET.…
The European Research Network for rare skin diseases (ERN-Skin) is launching a series of scientific webinars on rare skin diseases. Each 1-hour webinar will consist of a 30-minute presentation and a 30-minute discussion. The two upcoming webinars are: June 29th from 13.00 – 14.00 CET: Genetic discoveries in ichthyosis – Keith Choate Chaired by Juliette…