IS THERE A TREATMENT FOR FABRY DISEASE?

Up to some years ago, Fabry disease was treated symptomatically: disease progression in the various organs was contained pharmacologically and following an appropriate diet, while waiting for organ transplant (kidney, heart), when necessary and appropriate.

Recently, enzyme replacement therapy - ERT (intravenous injection of the missing enzyme) has become available for the treatment of Fabry disease, leading to promising results. New emerging therapies, such as gene therapy, may give further hope in trying to find a definitive solution to the disease.