3 results
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…
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…
The goal of this study is to investigate the efficacy and cost-utility of Oncokompas among patients with incurable cancer compared to care-as-usual. The main hypothesis is that use of Oncokompas will be superior to care as usual to improve patient…