Objective: To identify and map the available evidence on the implementation of public health
policies directed at individuals with rare diseases, and to compare the implementation of …

Background: Rare diseases are often serious, life-threatening and debilitating group of
disorders. Nowadays, there are approximately 8,000 rare diseases and it is estimated that …

This paper deals with the analysis of the costs, applied, for example, when treating specific
diseases–an important aid in prioritizing the process of resource allocation. In our review …

The fundamental criticism of the analyses of relations between the allocated sources into
healthcare system and general indicators of health status (represented by mortality) form a …

The article deals with one of the effects of health inequalities and gaps in access to
treatments for rare diseases, namely health-driven emigration. The purpose of the paper is …

The purpose of this study is to evaluate current legislative policies and regulations with
respect to the use of orphan drugs, to emphasize strengths and weaknesses of these …

ABSTRACT According to the European Commission, medical products for orphan diseases
are those which affect fewer than five persons out of every 10 000. Producers are reluctant to …

Enzyme replacement therapy (ERT) is the f rst-line treatment for Gaucher disease (GD). The
ERT Cerezyme (imiglucerase) was approved by the US Food and Drug Administration …

Статья посвящена этическим, социальным, психологическим и медицинским
проблемам, которые возникают в семьях, воспитывающих ребенка с редким …

Enzyme replacement therapy (ERT) is the first-line treatment for Gaucher disease (GD). The
ERT Cerezyme (imiglucerase) was approved by the US Food and Drug Administration …