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Notícias (Doenças raras)

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.

23andMe and Genentech to Analyze Genomic Data for Parkinson's Disease

23andMe, Inc., the leading personal genetics company, today announced an agreement with Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), to generate whole genome sequencing data f[...]

Record number of medicines for rare diseases recommended for approval in 2014

Number of medicines with new active substances continues to increase[...]

Scientific guideline: Guideline on the use of minimal residue disease as an endpoint in chronic lymphocytic leukaemia studies, draft: consultation open

The scope of this document is to describe the basis and regulatory requirements for the use of minimal residue disease (MRD) as an intermediate endpoint to predict clinical benefit in trials in chron[...]

Regional conference on Rare Diseases (2-3 December 2014, Prague)

The Consumers, Health and Food Executive Agency implements the EU Health Programme, the Consumer Programme and the Better Training for Safer Food initiative[...]

Advanced genomics for livestock breeding, health and welfare

Europe is home to a high proportion of the world's leading animal breeding organisations. Research being carried out by an EU-funded QUANTOMICS project is helping these breeders to remain competitive[...]

Cerdelga recommended for approval in type 1 Gaucher disease

New oral treatment option for rare debilitating genetic disease[...]

Scenesse recommended for rare disease that causes intolerance to sunlight

Patients involved in discussions on benefits and risks of a medicine at CHMP for the first time[...]

Distinguishing between Progressive Supranuclear palsy and Parkinson's disease

Progressive Supranuclear palsy (PSP) and Parkinson's Disease (PD) have overlapping symptoms but remain difficult to distinguish.[...]

Frontal deficits differentiate progressive supranuclear palsy from Parkinson's disease

These findings suggest that more severe and prominent 'frontal' cognitive deficits in patients with progressive parkinsonism would be helpful in predicting progressive supranuclear palsy rather than [...]

Scientific guideline: Draft concept paper on the revision of the guideline on the development of new medicinal products for the treatment of Crohn’s disease, draft: consultation open

The scope of this planned revision to the guideline on the development of new medicinal products for the treatment of Crohn's disease was extended to include adult as well as paediatric populations i[...]

Scientific guideline: Draft guideline on core SmPC for human plasma derived and recombinant coagulation factor IX products , draft: consultation open

This guideline describes the information to be included in the Summary of Product Characteristics (SmPC) for human plasma derived and recombinant coagulation factor IX products, which are indicated f[...]

Flt3 ligand does not differentiate between Parkinsonian disorders

No validated biomarkers are available for early and accurate diagnosis of multiple system atrophy and other parkinsonian disorders. It has been reported that flt3 ligand levels in cerebrospinal fluid[...]

Ketoconazole HRA recommended for approval in Cushing's syndrome

European Medicines Agency facilitates patients' access to a treatment of a rare hormonal disorder[...]

Success Stories - Reducing the risk of Alzheimer's disease and vascular dementia - World Alzheimer Day 21-09-2014

Dementia is a group of degenerative diseases typically affecting the cognitive abilities of the elderly population. According to the World Health Organization, the estimated proportion of the general[...]

Research Headlines - New hope for patients suffering from rare genetic disease

Very few of us have heard of Alpha-Mannosidosis. However, this rare genetic disease has affected the lives of hundreds of families across Europe and the world. Its first symptoms appear in early chil[...]


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