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Updated: Jan 26, 2026

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein
Published on: March 9, 2015
CFTR modulators reduce acute pancreatitis episodes in individuals with CFTR-related disorders: A case series
Mitchell L Ramsey1, Maria Corzo2, Rosara Bass2
1Division of Gastroenterology, Hepatology, and Nutrition; College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA; Division of Pediatric Pulmonology; Nationwide Children's Hospital, Columbus, OH, USA.
Abstract:
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators (CFTRm) reduce the frequency of recurrent acute pancreatitis (RAP) among individuals with pancreas sufficient cystic fibrosis (PS-CF), but the impact on RAP among individuals with CFTR-related disorder (CFTR-RD) is unknown. CFTRm are thought to be similarly effective in CFTR-RD because the mechanism of AP is identical to PS-CF. However, there are limited reports of CFTRm for CFTR-RD in the literature. We present the case histories of three individuals with RAP due to CFTR-RD who commenced treatment with CFTRm. Including 8.5 person-years of follow up, there have been no additional AP episodes during treatment and all individuals have tolerated treatment very well. This report indicates that CFTRm are effective in reducing AP frequency in some individuals with RAP due to CFTR-RD. Therefore, clinical trials to assess the effects of CFTRm for CFTR-RD are warranted, particularly for RAP where pharmaceutical treatments are lacking.
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