The road ahead appears promising
While there has certainly been significant progress made, that does not mean that there is not even farther to go. McColley points out that she is proud of how far research and treatment has come, but that she cannot pretend that there is not more that she is waiting for. She says, "while significant progress has been made with supportive therapies for cystic fibrosis, developing treatments that address the underlying genetic cause has been a challenge. We will need more analyses and longer-term data to see if this treatment can alter the disease and further extend the life expectancy of patients." Of course, this is a major goal and people across the field are joyed to have come so far.
For more information, the findings of the clinical trial have been published online at the New England Journal of Medicine.