engleski

Follow-up of enzyme replacement therapy in lysosomal diseases

Lysosomal storage diseases comprise a group of more than 40 disorders with a collective incidence of a approximately 1 in 7, 500 live births. In the last decade, a significant progress has been made not only in diagnostics but also therapy of this group of diseases. Currently, enzyme replacement therapy is a standard treatment for Gaucher disease, Fabry disease and mucopolysaccharidosis type I.

lysosomal diseases; enzyme replacement therapy

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