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Notizie (Malattie rare)

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.

Events - World Orphan Drug Congress Europe 2014 - 12-14 November 2014, Brussels

World Orphan Drug Congress is Europe's largest commercially-focused event for the rare disease research and advocacy. It is where pharma, biotech, patient groups and regulators come together to partn[...]

Events - Keystone Symposia: Precision Genome Engineering and Synthetic Biology - 11-15 January 2015, Big Sky, United States

This meeting gathers experts in development and application of synthetic biology and gene engineering fields to share ideas, methods, problems and solutions in a new era of investigational biology.[...]

European Medicines Agency looks to stimulate better use of genomic data and biomarkers in medicines development and safety monitoring

The European Medicines Agency (EMA) has released a draft concept paper today for public consultation outlining the key elements that will be developed in a guideline on good genomics biomarker practi[...]

Scientific guideline: Concept paper on good genomics biomarker practices, draft: consultation open

Genomic data have become important to evaluate efficacy and safety of a drug for regulatory approval. As a result, genomic information has been increasingly included in drug labels relevant for the b[...]

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 July 2014

This page provides an overview of the opinions adopted at the July 2014 meeting of the Committee for Medicinal Products for Human Use (CHMP) and other important outcomes.[...]

European Medicines Agency recommends approval of two new treatment options for rare cancers

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), at its July meeting, recommended granting marketing authorisations for two new medicines with novel mechan[...]

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[...]


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