3 results
Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).
To characterize safety, tolerability, pharmacodynamic effects, and pharmacokinetics of the oral sGC stimulator BAY 1021189 in addition to standard therapy for heart failure with reduced EF (HFrEF) over 12 weeks in patients with worsening chronic…
1: Compare the differences in the PRAGMA-CF score: the volume proportion of the lung with structural airways disease (%Dis), measured from chest CT images at 48 weeks between treatment arms 2: Compare the differences in PRAGMA-CF subscores: the…