Infant with cystic fibrosis has normal pancreas function and was missed by newborn screening after mom stayed on CFTR modulator therapy during pregnancy.
Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: Rationale and design of the CHEC-SC Study
Lumacaftor/ivacaftor reduces exacerbations in adults homozygous for Phe508del mutation with severe lung disease
CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials
GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1)
Disease Progression in Patients with Cystic Fibrosis Treated with Ivacaftor: Data From National US and UK Registries
Concentration of Fractional Excretion of Nitric Oxide (FENO): A Potential Airway Biomarker of Restored CFTR Function
Long-term improvement of lung clearance index in patients with mild cystic fibrosis lung disease: Does hypertonic saline play a role?