A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...

Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the ...

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is a combination medicine approved by the FDA in 2024. Alyftrek is approved to treat people ages 6 and older who have ...

A cell-penetrating nanobody repairs misfolded CFTR proteins in cystic fibrosis cell models, restoring function and boosting ...

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...

A new study from The Hospital for Sick Children (SickKids) reveals the process underlying protein organization on cell membranes, a finding which could pave the way for innovative cystic fibrosis ...

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...

Cystic fibrosis (CF) is a rare autosomal recessive disorder that affects numerous systems of the body. It is a complicated disease that differs from person to person. An autosomal recessive disorder ...

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved ...