NEWS

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![…]

EFPIA, EATRIS, ELIXIR, BBMRI, ECRIN statement on the role of research infrastructures to boost patient-centred research and innovation in Europe

The European Union has invested heavily in health research. Since the early 2000’s it supported the creation of European Research Infrastructures (pan-European organisations devoted to health research) like BBMRI, EATRIS, ECRIN, ELIXIR, and funded the Innovative Medicines Initiative (IMI), the world’s largest public private partnership (PPP) in health. The combination of PPPs such as the IMI and European infrastructures Read more about EFPIA, EATRIS, ELIXIR, BBMRI, ECRIN statement on the role of research infrastructures to boost patient-centred research and innovation in Europe[…]

UNO SGUARDO RARO (A Rare Glance)

The rare disease international film festival is the first and only film festival on rare diseases in Europe. It collects and promotes the best video works on rare diseases and social inclusion through an international competition and works with public and private partners, both national and international, the rare diseases community, disability advocates, entertainment and Read more about UNO SGUARDO RARO (A Rare Glance)[…]

RE(ACT) Congress 2020 and IRDiRC Conference, Berlin, Germany, 11th -14th March 2020

SAVE THE DATE!!! The BLACKSWAN Foundation and IRDiRC, the International Rare Diseases Research Consortium, are glad to announce the RE(ACT) Congress 2020 and IRDiRC Conference, Berlin, Germany, 11th-14th March 2020. This joint event will continue the RE(ACT) Congress series (6th edition) and IRDiRC Conference series (4th edition). It aims to bring together scientific leaders and experts and young scientists from a Read more about RE(ACT) Congress 2020 and IRDiRC Conference, Berlin, Germany, 11th -14th March 2020[…]

Rare disease perspectives in Central – Eastern Europe

On September 16th 2019, a workshop “Rare disease perspectives in Central – Eastern Europe”, twinned to the General Assembly of EJP RD, will be organized in Gdansk, Poland. The main aims of this conference are discuss challenges and opportunities of rare disease research in Central – Eastern Europe countries and areas of involvement within EJP Read more about Rare disease perspectives in Central – Eastern Europe[…]

EMA’s human medicines committee (CHMP) recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease

EMA’s human medicines committee (CHMP) has recommended the authorisation of Palynziq (pegvaliase), a new medicine for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease. Patients suffering from this disorder do not have the enzyme that breaks down phenylalanine, an amino acid which is found in most foods containing Read more about EMA’s human medicines committee (CHMP) recommends authorisation of new treatment for phenylketonuria, a rare inherited metabolic disease[…]

New results of #RareBarometer survey on patient’s preferences on rare disease data sharing & protection

A new EURORDIS-led article, “Share and protect our health data: an evidence based approach to rare disease patients’ perspectives on data sharing and data protection – quantitative survey and recommendations” has just been published in the Orphanet Journal of Rare Diseases. The peer-reviewed article is freely accessed, it was written in collaboration with Rebecca Dimond, Read more about New results of #RareBarometer survey on patient’s preferences on rare disease data sharing & protection[…]