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) ...
Chronic lung inflammation in cystic fibrosis (CF) often persists even after treatment with newly-approved gene therapies or small molecule CFTR modulators—an unresolved clinical paradox. A new study ...
Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...
A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
Dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) — an anion channel found on epithelial cells of various tissues — causes cystic fibrosis and plays an important role in ...
The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.
- 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 ...
Please provide your email address to receive an email when new articles are posted on . “[These findings] help clinicians be more supportive of a female with CF becoming pregnant,” Raksha Jain, MD, ...
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 ...
A cell-penetrating nanobody repairs misfolded CFTR proteins in cystic fibrosis cell models, restoring function and boosting ...
The FDA approved a once-daily oral combination of vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis (CF) in adults and children 6 years and up ...
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 ...
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