administering Alteplase (Cathflo) CFDI Canadian Dr. M. West Prospective study of enzyme 440 Oct 1 Provincial replacement therapy in 2016 Fabry disease Fabry disease Initiative sponsored LAL-CL06 Dr. M. West Study of Sebelipase enzyme 1 2017 Industry replacement therapy in sponsored lysosomal lipase deficiency FOS: Dr. M. West Registry collecting 72+ open Shire Fabry Outcome clinical data on Fabry HGT patients. Survey Fabry Registry Dr. M. West Registry collecting 76+ open Genzyme clinical data on Fabry patients. Gene Transfer Dr. M. West A gene therapy clinical 0 2021 CIHR Therapy in Fabry study directed towards sponsored Disease amelioration of Fabry disease in men using Lentivirus-transduced, autologous CD34+ stem cells will demonstrate safety and feasibility. REP-081 Dr. M. West A III clinical to 40 open determine the safety of a HGT new form of agalsidase-alfa enzyme replacement therapy in Fabry disease. CS4 Study: A Dr. K. The overall objectives are to 4 industry Phase 2, Tennankore evaluate the safety, Randomized, Dr. tolerability, Double Blind, Kiberd, Dr. pharmacokinetics, and Placebo Soroka pharmacodynamics of ISIS Controlled Study 416858 in ESRD patients on of the Safety, hemodialysis Pharmacokinetics, and of Multiple
administering Alteplase (Cathflo) CFDI Canadian Fabry disease Initiative Dr. M. West Prospective study of enzyme replacement therapy in Fabry disease 440 Oct 1 2016 Provincial sponsored LAL-CL06 Dr. M. West Study of Sebelipase enzyme replacement therapy in lysosomal lipase deficiency 1 2017 Industry sponsored FOS: Fabry Outcome Survey Dr. M. West Registry collecting clinical data on Fabry patients. 72+ open Shire HGT Fabry Registry Dr. M. West Registry collecting clinical data on Fabry patients. 76+ open Genzyme Gene Transfer Therapy in Fabry Disease Dr. M. West A gene therapy clinical study directed towards amelioration of Fabry disease in men using Lentivirus-transduced, autologous CD34+ stem cells will demonstrate safety and feasibility. 0 2021 CIHR sponsored REP-081 Dr. M. West A III clinical to determine the safety of a new form of agalsidase-alfa enzyme replacement therapy in Fabry disease. 40 open HGT CS4 Study: A Phase 2, Randomized, Double Blind, Placebo Controlled Study of the Safety, Pharmacokinetics, and of Multiple Dr. K. Tennankore Dr. Kiberd, Dr. Soroka The overall objectives are to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ISIS 416858 in ESRD patients on hemodialysis 4 industry
renal-program-annual-report-20152016.docx