Tag results:
cystic fibrosis
Pulmonary Cell News
Aceragen Announces $3.5 Million Development Award from Cystic Fibrosis Foundation to Progress Novel Therapy for CF-Related Exacerbations
[Aceragen, Inc.] Aceragen, Inc. announced that its wholly-owned subsidiary, Arrevus, Inc., has received an award from the Cystic Fibrosis Foundation for up to $3.5 million to support the clinical development of ACG-701, a proprietary formulation of sodium fusidate, as a treatment for cystic fibrosis-related pulmonary exacerbations.
Organoid News
Theratyping Cystic Fibrosis In Vitro in ALI Culture and Organoid Models Generated from Patient-Derived Nasal Epithelial Conditionally Reprogrammed Stem Cells
[European Respiratory Journal] Scientists exploited an innovative cellular approach allowing highly efficient in vitro expansion of airway epithelial stem cells through conditional reprogramming from nasal brushing of cystic fibrosis patients.
Endothelial Cell News
CFTR Limits F-Actin Formation and Promotes Morphological Alignment with Flow in Human Lung Microvascular Endothelial Cells
[physiological reports] Investigators tested the hypothesis that the cystic fibrosis transmembrane conductance regulator regulated endothelial actin cytoskeleton dynamics and cellular alignment in response to flow.
Organoid News
The Sodium/Glucose Cotransporters as Potential Therapeutic Targets for Cystic Fibrosis Lung Diseases Revealed by Human Lung Organoid Swelling Assay
[Molecular Therapy-Methods & Clinical Development] Scientists derived human proximal lung organoids from patient-derived pluripotent stem cells carrying disease-causing cystic fibrosis transmembrane conductance regulator mutations.
Organoid News
Eloxx Pharmaceuticals Reports Positive Topline Results from Monotherapy Arms of Phase II Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients
[Eloxx Pharmaceuticals, Inc.] Eloxx Pharmaceuticals, Inc. announced positive topline results from the monotherapy arms of its Phase II clinical trial of ELX-02 in Class 1 cystic fibrosis patients with at least one G542X nonsense allele mutation.
Intestinal Cell News
A Potential Treatment of Congenital Sodium Diarrhea in Patients With Activating GUCY2C Mutations
[Clinical and Translational Gastroenterology] Scientists investigated the effect of SSP2518 on guanylyl cyclase C (GCC)-mediated intracellular cyclic guanosine monophosphate levels and on GCC-mediated chloride secretion in intestinal organoids from three patients with distinct activating GCC mutations and from controls.