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Noticias (Enfermedades poco comunes)

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.

Advanced genomics for livestock breeding, health and welfare    europa.eu

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    europa.eu

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

Scenesse recommended for rare disease that causes intolerance to sunlight    europa.eu

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    epda.eu.com

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    epda.eu.com

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 guideline on core SmPC for human plasma derived and recombinant coagulation factor IX products , draft: consultation open    europa.eu

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[...]

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    europa.eu

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[...]

Flt3 ligand does not differentiate between Parkinsonian disorders    epda.eu.com

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    europa.eu

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    europa.eu

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    europa.eu

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[...]

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

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    europa.eu

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    europa.eu

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    europa.eu

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[...]

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

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[...]

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

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.[...]

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

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    xplorehealth.eu

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    europa.eu

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[...]


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