2 results
Approved WMOPending
The objectives of this are to establish the safety, tolerability, and preliminary efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in NHV and TED patients over a dose range of 3.0 to 20.0 mg/kg.
Approved WMORecruiting
Primary Objective: 1) To evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecundary Objective:1) To evaluate the efficacy of DTX301 in 3 response categories2) To evaluate the effect of…