Fabry Disease Study
Joseph M. Sanzari Children’s Hospital is one of two current sites nationwide pioneering research for new therapy for kidney disease.
Hackensack Meridian Joseph M. Sanzari Children’s Hospital was selected as one of the two current sites in the nation to be a part of a study, entitled “An Open-Label, Multinational Study of the Efficacy and Safety of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy Avr-Rd-01 for Treatment-Naïve Subjects With Classic Fabry Disease.
Kidney problems are common in individuals diagnosed with Fabry disease. Fatty molecules, known as globotriaosylceramide (Gb3 or GL-3), accumulate inside the patient’s cells, due to a lack of the enzyme alpha-galactosidase A. This can progress (if untreated) to kidney failure. The goal of the study is to provide a one-time therapy for the condition versus the current cadence of intravenous enzyme replacement therapy every other week. The trial treatment involves introducing a functional gene (GLA gene) to the patient and for the gene to continue to function and produce the desired protein (enzyme), alpha-galactosidase A.