Genetic dead ends: Why some cystic fibrosis patients can't access breakthrough drugs

Researchers have identified a subset of cystic fibrosis patients with severe liver damage who carry genetic mutations that make them ineligible for the latest CFTR-modulating therapies. The finding highlights a gap in precision medicine: while blockbuster CF drugs have transformed outcomes for many, a vulnerable population remains without effective pharmaceutical options, raising questions about drug development priorities and patient stratification.

Originaltitel: Characterization of CFTR mutations in people with cystic fibrosis and severe liver disease who are not eligible for CFTR modulators