4 results
The effectiveness an cost effectiveness of PPIs compared to standard phlebotomy therapy in the prevention of iron overload in patients with hereditary hemochromatosis.
The primary objective of the present study is to establish the histological effects of givinostat versus placebo administered over 12 months. The secondary objectives of this study are the following: - To establish the macroscopic muscle effects of…
Primary objective:* To establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects.Secondary objectives:* To assess the safety and tolerability of givinostat versus…
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.