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Initiation of CFTR Modulator
CFTR modulators restore function to the cystic fibrosis transmembrane conductance regulator (CFTR) protein, improving chloride ion transport across cell membranes.
CFTR modulators restore function to the cystic fibrosis transmembrane conductance regulator (CFTR) protein, improving chloride ion transport across cell membranes. Used for Cystic fibrosis (specific mutations dependent on modulator type).
At a glance
| Generic name | Initiation of CFTR Modulator |
|---|---|
| Also known as | 129Xe |
| Sponsor | Children's Hospital Medical Center, Cincinnati |
| Drug class | CFTR modulator |
| Target | CFTR (cystic fibrosis transmembrane conductance regulator) |
| Modality | Small molecule |
| Therapeutic area | Pulmonary/Genetic |
| Phase | FDA-approved |
Mechanism of action
CFTR modulators work by either correcting the trafficking and localization of defective CFTR protein to the cell surface (correctors) or enhancing the channel-opening activity of CFTR at the membrane (potentiators). This restores the ability of epithelial cells to properly regulate salt and water balance, reducing the thick mucus secretions characteristic of cystic fibrosis.
Approved indications
- Cystic fibrosis (specific mutations dependent on modulator type)
Common side effects
- Transaminase elevation
- Headache
- Nausea
- Diarrhea
Key clinical trials
- Non-contrast Lung Perfusion Mapping Applied for New Insights in Cystic Fibrosis (PHASE4)
- Hyperpolarized Imaging for New Treatments (PHASE4)
- CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
- Effectiveness of CFTR Modulators According to Co-therapy
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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