2 results
Approved WMOCompleted
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation
Approved WMOCompleted
Primary objective:Determine the short- and long-term safety and tolerability of single and multiple doses of NI006 in subjects with ATTR-CM by evaluating adverse events (AE) and serious adverse events (SAE) and changes in laboratory parameters (…