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)
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…
The primary objective is to evaluate the feasibility and adherence of 6 weeks of rehabilitation with the SEA in participants who underwent TL. The secondary objective is to examine if the SEA is an effective tool to improve swallowing function and…
Evaluate the long-term safety and effectiveness of two dose levels of orally administered BCX7353 in subjects with HAE