Treatment with lumacaftor-ivacaftor (Orkambi) was tolerated well and maintained its efficacy in children aged 6-11 years out to 120 weeks, according to long-term trial data. Previous trials have demonstrated success with the combination therapy in children homozygous for the F508del-CFTR mutation out to 24 weeks. “The safety and efficacy of the lumacaftor-ivacaftor combination has not ...
Benefits of cystic fibrosis combination therapies maintained in the long-term: trials
23 Feb 2021