Open access
Sep 2026
In Vitro Responsiveness of 417 CFTR Variants to Vanzacaftor/Tezacaftor/Ivacaftor.
The findings may inform people with cystic fibrosis and caregivers pursuing off-label access to modulator therapies when genotype-based eligibility is limited and highlight remaining variants that may require alternative therapeutic strategies.
H. Bihler, Andrey Sivachenko, Priyanka Bhatt et al.
· Journal of Cystic Fibrosis · 0 citations