A Therapy for Most with Cystic Fibrosis.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 31978337.
- Also identified by DOI 10.1016/j.cell.2019.12.032.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.
Medical subject headings
- Aminophenols
- Benzodioxoles
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Indoles
- Pyrazoles
- Pyridines
- Quinolines