Newly adopted Marketing Authorisation Decisions (last six months) The Union Register lists all medicinal products for human and veterinary use as well as orphan medicinal products that have received a marketing authorisation by the @EU_Commission through the centralised procedure. Check out the full list here.
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The Economic, social and cultural rights new report of the UN highlights the status quo and future needs of persons living with rare diseases. « Rare diseases often attract stigma and discrimination, and many persons living with a rare disease find themselves excluded from participation in employment and from integrating fully and productively into society ».
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Nitisinone is probably the biggest source of hope for alkaptonuria (AKU) patients right now. Research efforts under the DevelopAKUre project are bringing its commercialisation within reach. DevelopAKUre (Clinical Development of Nitisinone for Alkaptonuria), the project investigating nitisinone supported by the AKU Society and coordinated by the Royal Liverpool and Broadgreen University, is close to completing its…
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Speaking in Brussels, Yann Le Cam, CEO of Eurordis, the European organisation for rare diseases, said, “We have gone from near ignorance to the recognition of rare diseases as a public health priority in Europe.” He added, “The landscape has changed drastically; expertise and innovative technologies that can potentially benefit people living with a rare…
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The United Nations Office of the High Commissioner for Human Rights (OHCHR) has made reference to persons living with a rare disease within its recently published annual report to the UN Economic and Social Council (ECOSOC). The 2019 report focuses on the topic of Universal Health Coverage (UHC), and looks at how the human rights framework…
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European Joint Programme on Rare Diseases serves as official partners for the 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020) taking place at the Stockholmsmässan Congress Center in Stockholm, Sweden on 15 – 16 May 2020. The overarching theme for the conference is: The rare disease patient journey in 2030. More info…
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As part of the European Biotech Week, EuropaBio will host a panel discussion on the 23rd of September in Brussels. Experts from the biotech financing ecosystem in Europe will discuss the future for access to finance in the EU and present different approaches to keep up in the race with biotech front-runners such as China…
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On September 18th and 19th in Brussels, PARADIGM, PFMD and EUPATI will organise the Patient Engagement Open Forum. The Forum aims to provide a holistic perspective of patient engagement, the landscape and actors, and foster collaboration and co-creation while breaking down fragmentation and silos that are often present in patient engagement work. The agenda of the two days…
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« European Initiative Targets Diagnosis, Treatment of Rare Diseases » article has just been released on ALS NEWS Today! Check it out! <<A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such…
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Representatives from paediatric networks, research infrastructures (RIs), European Reference Networks (ERNs), charities, patients’ associations, Young Persons Advisory Groups (YPAGs), companies’ federations, Governments and regulatory Authorities had a very productive discussion during the stakeholders roundtable held in the framework of the 3rd General Assembly of EPTRI (European Paediatric Translational Research Infrastructure) in Madrid, Spain, on March…
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