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News (Rare diseases)

This section on health in the EU has been created as a trustworthy gateway to a wide range of information and data on health-related issues and activities at both European, national and international level. The content is produced by the European Commission, the Member States of the EU and the European Economic Area (EEA), plus EU candidate countries; by international organisations; and by pan-European non-governmental organisations in the area of public health.

Registration opens for workshop on clinical trial design in neuromyelitis optica to facilitate global medicine development in a rare and serious disease

Registration is now open for a regulatory workshop on clinical trial design in neuromyelitis optica and spectrum disorders at the European Medicines Agency on 10 October 2014.[...]

New resource for educators: Discussion games: Orphan drugs

Game to engage in a dialogue | How are drugs developed? Orphan drugs are medicines which have been developed to diagnose and treat rare diseases. Although they can be beneficial for millions of peop[...]

Events - International Rare Disease Research Consortium Shenzhen Conference - 7-9 November 2014, Shenzhen, China

The second conference organized by IRDiRC in collaboration with the BGI is gathering stakeholders active in the field of rare disease from across the globe. This conference aims to raise awareness of[...]

European Medicines Agency and US Food and Drug Administration release joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have released today a draft joint proposal to facilitate the clinical investigation of new medicines for t[...]

Test your knowledge with the new ECG quiz

An 18-year-old female attends a routine occupational medical in order to join the British Army. She represents her school and region in cross-country running and hockey. She is asymptomatic with no f[...]

European Medicines Agency recommends approval of a locally targeted treatment for ulcerative colitis and Crohn’s disease

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended granting a marketing authorisation for Entyvio (vedolizumab) for the treatment of adult patients [...]

Joint EMA/FDA/MHLW-PMDA orphan medicinal product workshop takes place today

A workshop on orphan product designation and grants is taking place today at the European Medicines Agency (EMA), jointly organised by the EMA, the US Food and Drug Administration (FDA) and for the f[...]

Safe treatment for AK, a rare eye disease

The ODAK ('Orphan Drug for Acanthamoeba Keratitis') project is working to develop a safe and effective treatment for Acanthamoeba keratitis (AK), a rare infectious eye disease which causes severe deb[...]


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