1. Real-Life Experience with CFTR Modulators Shows Correction of LAD-IV Phenotype in Cystic Fibrosis.
- Author
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Montresor, Alessio, D'Ulivo, Beatrice, Preato, Sara, Farinazzo, Alessia, Pintani, Emily, Chiara, Elena Dalla, Torroni, Lorena, Verlato, Giuseppe, Boscia, Silvia, Pisano, Laura, Mangone, Giusi, Ricci, Silvia, Azzari, Chiara, Taccetti, Giovanni, Terlizzi, Vito, Cipolli, Marco, Melotti, Paola, Sorio, Claudio, and Laudanna, Carlo
- Subjects
DRUG accessibility ,RHO GTPases ,BONE marrow ,CYSTIC fibrosis ,CYSTIC fibrosis transmembrane conductance regulator ,CHLORIDE channels ,MUCOCILIARY system - Abstract
This document summarizes a study on the effects of CFTR modulators on patients with cystic fibrosis (CF) and leukocyte adhesion deficiency type IV (LAD-IV). The study found that CFTR modulators improved certain cellular functions in CF patients, suggesting that they may correct both CF and LAD-IV phenotypes. However, there were some patients who showed improvement in CF symptoms but not in LAD-IV characteristics, indicating the need for further research. The study also suggests that measuring certain cellular markers could be a useful method for monitoring patients in the long term. [Extracted from the article]
- Published
- 2024
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