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1000
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Abstract/Summary
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Cystic fibrosis (CF) has a complex and chronic manifestation, with the severity and types of symptoms differing widely from person to person depending on their specific genetic mutation, their environment and their genetic background. Individuals with CF have treatment plans aimed at ameliorating the debilitating symptoms of their disease, including difficulties in breathing, recurrent lung infections and digestive disorders. The year 2012 marked a turning point for certain eligible CF patients — KALYDECO™ was approved as a therapy for a small group of CF patients exhibiting the G551D mutation [1] and eight other rare CFTR mutations in the CFTR gene.
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