4 results
- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…
Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…
To determine the most effective drug (iNO or IV sildenafil) for the treatment of PH in newborns with CDH and to reach evidence based dosing of this drug. Subsequently this drug will be added to the international guidelines for the treatment of…
To investigate whether early treatment with levodopa has a delayed beneficial effect on PD symptoms and functional health, and improves the ability to (maintain) work, reduces the use of (informal) care, caregiver burden, and costs. Additionally,…