CLINICAL AND ECONOMIC EFFICACY OF USING AGALSIDASE ALPHA IN PATIENTS WITH FABRY DISEASE
Rare (orphan) diseases are diseases that occur with a frequency of 1 case per 10 thousand people. Most orphan diseases occur in childhood and about 30% of children with rare diseases do not live to 5 years. In Kazakhstan, the total number of identified patients suffering from hereditary diseases (including Fabry) is less than 1%