3 results
Primary objective:Determine the incidence rate of inhibitor formation in PUPs with severe and moderately severe hemophilia A during the first 50 exposure days (EDs) to starting with a once weekly prophylactic regimen together with the minimization…
The purpose of this study is to compare the effectiveness of treatment Arm A and Arm B by measuring the event free survival (EFS) and pathological complete response (pCR). EFS is defined as the time from the first treatment dose that the patient…
Primary:•To compare the effect of venglustat with standard of care Fabry therapies on left ventricular mass index over 18 months in participants with Fabry disease and left ventricular hypertrophySecondary:•To evaluate the effect of venglustat on…