Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the gene that encodes the transmembrane conductance regulator of CF (CFTR), a protein that acts as a chloride channel. Viscous secretions resulting from altered transport of chloride and sodium progressively lead to dysfunction of the exocrine glands of multiple organic systems, and mainly affect the respiratory system but also the pancreas, bile ducts, sweat glands and genitourinary system. The most common manifestations of the disease are exocrine pancreatic insufficiency in about 85-90% of cases and severe chronic obstructive pulmonary disease, the leading cause of morbidity and mortality in these patients.
Lumacaftor; Ivacaftor; Cystic fibrosis; Minority disease
Programa d'Harmonització Farmacoterapèutica. Lumacaftor/ivacaftor (Orkambi®) per al tractament de la fibrosi quística. Barcelona: Servei Català de la Salut; 2020.
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