3 results
The objectives are to:A. Identify subjects with VWD who may benefit from prophylaxis by determining patterns of bleeding prior to evaluation for enrollmentB. Study the effect of prophylaxis on bleeding frequencyC. Establish optimal treatment…
The main objective of this study is to determine whether lowering estrogen and progesterone levels with leuprorelin decreases liver growth rates in pre-menopausal women with severe PLD.Secondary objectives are to assess in these women the effect of…
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…