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Chiara Barzan

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Articles

Gaucher Disease: A Glance from a Medicinal Chemistry Perspective

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By Filippo Prencipe, Chiara Barzan, Chiara Savian, Giampiero Spalluto
| Wiley Online Library Verified
Rare diseases are particular pathological conditions affecting a limited number of people and few drugs are known to be effective as therapeutic treatment. Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase, belongs to this class of disorders, and it is considered the most common among the Lysosomal Storage Diseases. The two main therapeutic approaches are the Enzyme Replacement Therapy (ERT) and the Substrate Reduction Therapy (SRT).
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