Article ; Online: Cystic Fibrosis Modulator Therapies.
2022 Volume 74, Page(s) 413–426
Abstract: Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction syndrome, liver dysfunction, and other disorders. Traditional therapies ... ...
Abstract | Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction syndrome, liver dysfunction, and other disorders. Traditional therapies focused on the treatment or prevention of damage to each organ system with incremental modalities such as nebulized medications for the lungs, insulin for diabetes, and supplementation with pancreatic enzymes. However, the advent of highly effective modulator therapies that target specific cystic fibrosis transmembrane conductance regulator protein malformations resulting from individual genetic mutations has transformed the lives and prognosis for persons with CF. |
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MeSH term(s) | Humans ; Cystic Fibrosis/drug therapy ; Cystic Fibrosis/genetics ; Prognosis ; Mutation ; Diabetes Mellitus ; Aminophenols/therapeutic use |
Chemical Substances | Aminophenols |
Language | English |
Publishing date | 2022-08-16 |
Publishing country | United States |
Document type | Journal Article ; Review ; Research Support, Non-U.S. Gov't |
ZDB-ID | 207930-6 |
ISSN | 1545-326X ; 0066-4219 |
ISSN (online) | 1545-326X |
ISSN | 0066-4219 |
DOI | 10.1146/annurev-med-042921-021447 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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