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02/01/1900
Artículos

The aim of the author is to discuss special issues of rare diseases, with emphasis on circumstances present in Hungary, including those leading to the foundation of the non-governmental organization, the Hungarian Federation of People with Rare and Congenital Diseases. The author briefly reviews the most important findings of current international surveys which have been […]

02/01/1900
Artículos

Few areas of paediatric care are as susceptible to the revision of lines of action, and require such professional training, as the care of the child with a rare disease (RD) and his/her family, especially if there are malformations. The lifelong impact, which many RDs entail, make continuous monitoring of the child compulsory, centred both […]

02/01/1900
Artículos

The treatment of patients with rare diseases is a challenge for all those involved, since it frequently requires decisions on the basis of uncertainty. Uncertainty is of particular importance for the risk/benefit assessment of therapeutic interventions which “normally” imposes extensive requirements on the quality of evidence through explicit specifications. These requirements concern the assessment of […]

02/01/1900
Artículos

The importance of rare disease is appreciated by all parties and tremendous effort is made to increase the knowledge about the individual disorders and improve the care of affected patients. Political initiatives on a European level aim to improve the structure of medical care for patients with rare diseases in each member state. The provided […]

02/01/1900
Artículos

Under the influence of patients’ associations, rare diseases have gradually become recognised as a public health issue. A first Rare Diseases National Plan, from 2005-2008, endeavoured to facilitate diagnosis and access to care. A second Plan, for 2011-2014, aims to improve patient management and develop research and international cooperation. The Rare Diseases Platform provides an […]