2 results
Approved WMOPending
To define and implement an uniform initial evaluation and stratification on adult patients with LCH, to uniform the treatment of adult patients with LCH and to improve treatment results with respect to survival, therapy response, prevention of…
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…