2 results
Approved WMOCompleted
Main objectivePart A: To evaluate the safety and efficacy of treatment with VX-371, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are *12 years of age.Part B: To evaluate the safety…
Approved WMOCompleted
To evaluate the efficacy, safety, tolerability, in subjects with ALS following the oral administration of study drug (active or placebo capsules).