3 results
The primary efficacy variable is the change in PV from baseline as assessed by 3 D ultrasonography after 78 weeks of double-blind treatment with OM 20-40 mg daily compared to ATE 50-100 mg daily.Secondary efficacy variables:Change from baseline PV…
The FAST Therapy Trial is a prospective trial of patients with a new diagnosis of fetal SVA aimed to compare the impact of different perinatal treatment strategies from the time of SVA diagnosis to birth or death1) The primary outcome will be the…
Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To investigate the pharmacokinetics (PK) of risdiplam and metabolites as appropriate.Secondary ObjectiveThe secondary…