2 results
Approved WMORecruiting
The objectives of this are to establish the safety, tolerability, and efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in HV and TED patients over a dose range of 3.0 to 20.0 mg/kg.
Approved WMOPending
The primary objective of this study is to evaluate the performance of the Navigate Anti-AAV9 Antibody Assay using serum specimens in subjects with SMA in Novartis clinical studies COAV101B12301 *STEER* and COAV101B12302 *STRENGTH*.