3 results
The goal of this study is to investigate the efficacy and cost-utility of Oncokompas among patients with incurable cancer compared to care-as-usual. The main hypothesis is that use of Oncokompas will be superior to care as usual to improve patient…
We aim to prevent or reverse developmental regression in patients with Kleefstra syndrome. We hypothesise that the regression we observed is the result of a psychotic disorder, which isnot properly recognised by clinicians and often not treated…
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…