Longing for a Cure
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From the Cystic Fibrosis Gene to a Drug
While Alexis was successfully treated with drugs known to work on her condition, what about people who have genetic diseases for which there is no effective treatment?
In this video, you'll meet cystic fibrosis patient Michael McCarrick and learn about the challenges, time lines, and costs involved in developing drug therapies for genetic diseases. While safe and effective therapeutics may be on the horizon—if only for some patients—true breakthroughs remain few and far between. Click on the left to watch the video.
After watching the video, list some of the steps that led scientists to the development of Kalydeco. What role have patients played in this process, and what are the risks to these individuals?
