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cystic fibrosis

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.

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.

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.

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.

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.

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.

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