2 results
Approved WMOCompleted
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
Approved WMORecruiting
Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32…