A serious consideration about the wise use of the new, highly efficient, very expensive, treatment for cystic fibrosis (Trikafta©/Kaftrio©)
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
