2 results
Approved WMOCompleted
Primary: Longterm safety and tolerability.Secondary: Time to 1st flare, number and severity of flares, efficiacy in treating flares, effect on inflammatory markers, immunogenicity, PK.
Approved WMOCompleted
To assess the ability of MRI techniques to detect early stages of lipid accumulation in the liver of ASMD patients with the chronic visceral subtype compared to values of age-, sex- and BMI-matched healthy subjects.