4D-310 demonstrated clinically meaningful cardiac endpoint improvements through 12-24 months in contractility (echocardiography), peak VO2 (cardiopulmonary exercise testing) and/or cardiac quality of life (KCCQ) in all five evaluable patients Cardiac biopsies were obtained from a patient at week 6 and […]
Tag: 4DMT
4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy
Initiated single non-human primate (NHP) safety study evaluating intravenous 4D-310 combined with rituximab/sirolimus (R/S) immunosuppressive regimen Amended INGLAXA protocol to minimize risk of atypical hemolytic uremic syndrome (aHUS) associated with intravenous (IV) AAV dosing, including addition of R/S immunosuppressive regimen […]