2 results
Approved WMOCompleted
Part A: Determine safety and tolerability of FTX-1821 in healthy subjects. Part B and C: Determine safety and tolerability of FTX-1821 in FSHD patients.
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