2 results
Approved WMORecruiting
Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI for the total group (both prenatal and postnatal diagnosis) and the prenatal diagnosis group, which is needed to…
Approved WMORecruiting
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…