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Simplifying CFTR modulation therapy in cystic fibrosis

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When The Archivist started practicing paediatrics, the predicted age of survival for a child with cystic fibrosis (CF) was 15 years of age. Treatment was pancreatic enzyme supplementation, physiotherapy for airway clearance and antibiotics. By the1990s lung transplant, inhaled tobramycin and DNAse became available. Once the CF protein and CF genes were identified, median predicted survival was 44 years with 53% patients surviving over 18 years. The main problem is a defect of the CF transmembrane conductance regulator (CFTR) protein. In the 2020s CFTR modulators have had a major impact on CF management in some cases doubling the survival rate. …

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