The U.S. Food and Drug Administration approved Kalydeco® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.
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May 3, 2023
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4 min read
Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.
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Sept. 2, 2022
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3 min read