Gene Therapy – a phase II study is recruiting in Perth and Melbourne
You may have seen in the news (Channel Seven Perth) something about Gene Therapy in Fabry patients? To watch the Channel Seven Perth story video Press Here The company conducting the trial is AVROBIO. Their new study is assessing the efficacy and safety of a new gene therapy treatment AVR-RD-01.
Stem cells are special cells from the blood that can mature into many different cell types. In this study, these cells are collected from the patient, genetically modified and then returned to the patient by Intravenous (IV) infusion. The modified cells may then secrete the missing Alpha-galactosidase A (AGA) enzyme, removing the need for enzyme replacement therapy (ERT). To read about the new clinical trial Press Here
Further details can be discussed with your Fabry Clinic