The drug called Kalydeco was approved by the FDA today. This is the first drug that aims to target the underlying cause of Cystic Fibrosis. Our community has been so excited about the news of this drug. It does only aim to treat the cause of CF in people with a specific cf mutation (not Madeline's). Even though this exact drug will not help Madeline, this is the beginning of finding even more drugs to treat her mutation.
Kalydeco is an oral pill that is given by mouth. It aims to treat only 4% of CF patients with a a specific mutation. There are already two drugs in the pipeline set to go to the FDA in a few years(we hope) to help treat the most common cf mutation(madeline's mutation) delta 508.
We have so much hope that more GOOD things will come within the CF community. My eyes are watering at how this news will surely impact so many CF people for years to come...especially as we see more drugs become approved.
Thank you to everyone who has given a dollar, a cent, or their time to Madeline's cause....Today, you can see how it has paid off.
To read more about this new drug development ....click here. Another article here.
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