Author Archives: Alice Melao

Gene Therapy AVR-RD-01 Effectively Prevents Buildup of Toxic Gb3 in Fabry Patients, Interim Data Show

Avrobio’s investigational gene therapy AVR-RD-01 shows potential in promoting durable beneficial effects to treat Fabry disease, with enhanced therapeutic activity compared with the standard-of-care enzyme replacement therapy, according to preliminary clinical data. These findings were reported after the evaluation of the first eight patients with Fabry disease who…

LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows

Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and monitor Fabry disease, rather than only using GLA mutational status, researchers suggest. The study, “Mutations in the GLA Gene and LysoGb3: Is It Really Anderson-Fabry Disease?,” was published in the International Journal…