SpliSense is currently conducting a Phase IIb trial with its antisense oligonucleotide therapy.

KB407’s positive set of results presented at ATS 2026 follows a set of similar positive results that were announced for the first two cohorts in December 2024.

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...

Please provide your email address to receive an email when new articles are posted on . Patients from minority racial and ethnic groups with cystic fibrosis are less likely to be eligible for cystic ...

Vertex Pharmaceuticals Incorporated VRTX is a clear leader in the global cystic fibrosis (CF) market. The company has built a ...

A recent retrospective study published in CHEST evaluated the effects of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use on pregnancy outcomes in individuals with cystic ...

Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...

The availability of a simple, robust and non-invasive in vitro airway model would be useful to study the functionality of the cystic fibrosis transmembrane regulator (CFTR) protein and to personalize ...

Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...