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 WMORecruiting
To evaluate safety, tolerability pharmacokinetic profile; to explore pharmacodynamics; to evaluate major effects of Guanabenz on quantitative MRI and clinical parameters in pediatric patients with VWM; explore biomarkers in blood and cerebrospinal…