4 results
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
The primary hypotheses in this study is that a cangrelor infusion will maintain target levels of platelet inhibition (> 60% inhibition) after discontinuation of a thienopyridine (clopidogrel or ticlopidine) in patients waiting for surgery.
Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…
To demonstrate the feasibility to induce an effective, predictable and sustained decrease in FGF23 level in CKD stage I-IV patients, without inducing hypophosphatemia using a stepped treatment regimen aiming at restricting phosphate uptake.