2 results
Approved WMOCompleted
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…
Approved WMOCompleted
In patients with symptomatic New York Heart Association (NYHA) Class II/III/IV dilated cardiomyopathy (DCM) in which mutations in the gene encoding the lamin A/C protein (LMNA) have been implicated:Primary Objective:* NYHA Class II/III patients only…