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CFTR Modulator Therapy in an Individual With Cystic Fibrosis Caused by a N1303K CFTR Variant and Infected With Mycobacterium abscessus .

Authors :
Elson EC
Capel P
Haynes J
Duehlmeyer S
Fischer M
Escobar H
Source :
The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG [J Pediatr Pharmacol Ther] 2022; Vol. 27 (4), pp. 396-399. Date of Electronic Publication: 2022 May 09.
Publication Year :
2022

Abstract

This report describes a case of a 15-year-old male with cystic fibrosis caused by N1303K and Q493X cystic fibrosis transmembrane conductance regulator (CFTR) protein variants. In this case, CFTR modulators including tezacaftor-ivacaftor and subsequently elexacaftor-tezacaftor-ivacaftor were used and resulted in clinical stability and improvement.<br />Competing Interests: Disclosures. The authors declare no conflicts or financial interest in any product or service mentioned in the manuscript, including grants, equipment, medications, employment, gifts, and honoraria. The authors had full access to all patient information in this report and take responsibility for the integrity and accuracy of the report.<br /> (Copyright. Pediatric Pharmacy Association. All rights reserved. For permissions, email: mhelms@pediatricpharmacy.org 2022.)

Details

Language :
English
ISSN :
1551-6776
Volume :
27
Issue :
4
Database :
MEDLINE
Journal :
The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG
Publication Type :
Academic Journal
Accession number :
35558347
Full Text :
https://doi.org/10.5863/1551-6776-27.4.396