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Hi, I have been waiting 8 years for Ivacaftor to be approved for residual function CFTR mutations. The day finally came on May 17th and soon after, we had my son signed up and taking his first dose. We did this with a little trepidation based on the results of a small N-of-1 study...
The recent news articles about two big CF drugs not making it to market because of the FDA have left me a bit nervous. I have to wonder what impact this will have on the pharmas that have products in the pipeline.
I believe in safety first but it is frustrating to see setbacks like this with...
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