7 results
The aim is to assess the long-term safety of topical use of ZORBLISA in patients with Epidermolysis Bullosa (EB).
The primary objective is to compare the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, RecessiveDystrophic, or Junctional non Herlitz Epidermolysis Bullosa.The primary endpoint is the complete closure of the…
Primary objectives1A. To evaluate the effect of maximal LDL-C reduction by Evolocumab on top of high intensity lipid-lowering therapy , initiated immediately after invasive ACS treatment on functional impairment of non-infarct related artery (non-…
Primary:• To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH Secondary• To characterize PK of ravulizumab SC• To characterize PD of ravulizumab SC• To characterize immunogenicity of ravulizumab SC• To…
Main objective:To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) profile.Secondary objectives:• To assess the efficacy of…
To evaluate the efficacy of ravulizumab compared with placebo to reduceproteinuria in adult participants with LN or IgAN.
Primary objective: To evaluate the effect of ravulizumab compared with placebo on amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) score in adult patients with amyotrophic lateral sclerosis (ALS).Secondary objectives:1. To…