NEWS

Report of the United Nations High Commissioner for Human Rights states the needs in rare diseases

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”.

New AKU treatment comes closer to commercialisation

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 Read more about New AKU treatment comes closer to commercialisation[…]

Call for expression of interest to recruit an Ethics Advisor to the European Joint Programme on Rare Diseases

The EJP RD is looking for an Ethics Advisor, apply for the position! The Advisor will be in charge of verifying the application of EU ethical/legal/regulatory framework within the projects that will be financed within and by the EJP RD. A dedicated work package with 15 deliverables was created. Foundation GIANNI BENZI leads the activities Read more about Call for expression of interest to recruit an Ethics Advisor to the European Joint Programme on Rare Diseases[…]

Tackling rare diseases a ‘European success story’

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 Read more about Tackling rare diseases a ‘European success story’[…]

United Nations human rights body stresses the need to address rare diseases within Universal Health Coverage

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 Read more about United Nations human rights body stresses the need to address rare diseases within Universal Health Coverage[…]

10th European Conference on Rare Diseases & Orphan Products (ECRD2020)

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 Read more about 10th European Conference on Rare Diseases & Orphan Products (ECRD2020)[…]

Patient Engagement Open Forum

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 Read more about Patient Engagement Open Forum[…]

Daria Julkowska new interview on the EJP RD project!

“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 Read more about Daria Julkowska new interview on the EJP RD project![…]

EPTRI EU STAKEHOLDERS ROUNDTABLE SUMMARY REPORT RELEASED!

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 Read more about EPTRI EU STAKEHOLDERS ROUNDTABLE SUMMARY REPORT RELEASED![…]