AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Overview of clinical data from phase 1 and phase 2 studiesdoi:10.1016/j.ymgme.2020.12.259Mark Thomas aKathleen Nicholls bBenedict Carnley aMirjam Trame cChris Mason c...
AVR-RD-01 lentiviral gene therapy reduces Gb3 substrate in endothelial cells of renal peritubular capillaries in a previously untreated classic Fabry disease male patientdoi:10.1016/j.ymgme.2019.11.416Birgitte VolckShari FalletMark ThomasBenedict Carnley...