5 results
The primary efficacy variable is the change in PV from baseline as assessed by 3 D ultrasonography after 78 weeks of double-blind treatment with OM 20-40 mg daily compared to ATE 50-100 mg daily.Secondary efficacy variables:Change from baseline PV…
The study objectives are to determine the incidence rate of FVIII inhibitors, frequency of adverse events (AEs), and serious adverse events (SAEs) associated with the use of CSL627, to evaluate the PK of 50 IU/kg CSL627, and to evaluate the efficacy…
To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder
To evaluate the efficacy of rVIII-SingleChain in the treatment of major andminor bleeding events based on the investigator*s 4-point assessment scale
Investigating the long-term safety of personalized cholic acid treatment in patients with defects in bile acid synthesis based on clinical and biochemical parameters.