Waltham – In recent years the study on the origin of genetic diseases has opened the door to new therapies that were unthinkable a few decades ago, when assuming to treat a genetic disease with an oral medication would at least make people smile. Some of these diseases have genetic alterations for which the altered substance that is produced by the body can actually still be used by the body with the help of drugs. This is the result of a worldwide study on Fabry disease, published on the New England Journal of Medicine. Prof. Claudio Feliciani, professor of the Department of Clinical and Experimental Medicine, University of Parma and Director of the Dermatology Clinic of the University Hospital of Parma, was part of the research tema. The study proved that an oral drug similar to a sugar has “chaperonical properties”, i.e. by binding to the dysfunctional enzyme, it rehabilitates its endocellular scavenger function. (9colonne)
