4 results
To evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftormonotherapy through 8 weeks of treatment in subjects with cystic fibrosis (CF)who are heterozygous for the F508del mutation on the CF transmembraneconductance regulator (…
To evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Primary Objective1. To evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1Secondary Objectives1. To assess target engagement of losmapimod tablets in blood and skeletal muscle over long term dosing2…
Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease progression assessed by reachable workspace quantification of total relative surface area (RSA) Q1-Q5 with 500 g wrist…