A combination therapy for cystic fibrosis.
Where this comes from
- Record sourced from PubMed, PMID 26406363.
- Also identified by DOI 10.1016/j.cell.2015.09.003 and PMC identifier 4865247.
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Abstract
The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.
Medical subject headings
- Aminophenols
- Aminopyridines
- Benzodioxoles
- Cystic Fibrosis
- Quinolones