Results 121 to 130 of about 1,231,622 (330)

A retrospective cohort study evaluating the growth and clinical outcomes of preterm and term infants with cystic fibrosis during the first 11 years of life

JPGN Reports, EarlyView.
Abstract Objectives Infants with cystic fibrosis (iwCF) have lower birth weights than others. That influences nutritional and pulmonary outcomes. It was reported that 86.0% of iwCF were born full‐term and 14.0% preterm. Data on the role of gestational age in growth and clinical outcomes of people with cystic fibrosis (pwCF) is limited.
Hebah Reda, Sanjidah Ahmed, Julie Sturza, Samya Z. Nasr   +3 more
wiley   +1 more source

Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

iScience
Summary: Cystic fibrosis (CF) is a life-shortening autosomal recessive disease, caused by loss-of-function mutations that affect the CF transmembrane conductance regulator (CFTR) anion channel.
Lucia Nicosia, Iwona Pranke, Roberta V. Latorre, Joss B. Murray, Lisa Lonetti, Kader Cavusoglu-Doran, Elise Dreano, James P. Costello, Michael Carroll, Paola Melotti, Claudio Sorio, Isabelle Sermet-Gaudelus, Martina F. Scallan, Patrick T. Harrison   +13 more
doaj   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis

The Laryngoscope, EarlyView.
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Amy Lin, Makanko Komara, Isaac Kistler, Zheng Hong Tan, Meredith Lind, Kavitha Kotha, Tendy Chiang   +6 more
wiley   +1 more source

P-84 HAVE CFTR MODULATORS CHANGED THE NEED FOR LIVER TRANSPLANTATION AMONG PATIENTS WITH CYSTIC FIBROSIS? AN ANALYSIS OF THE UNOS DATABASE

Annals of Hepatology
Conflict of interest: No Introduction and Objectives: The impact of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators on the natural history of liver disease is unknown.
MANUEL MENDIZABAL, Claire Cywes, Nicolas Muñoz, Amay Banker, Christian Bermudez, Denis Hadjiliadis, Peter Abt   +6 more
doaj   +1 more source

Pharmacologic improvement of CFTR function rapidly decreases sputum pathogen density, but lung infections generally persist

The Journal of Clinical Investigation, 2023
Background Lung infections are among the most consequential manifestations of cystic fibrosis (CF) and are associated with reduced lung function and shortened survival.
David P. Nichols, Sarah J. Morgan, Michelle Skalland, Anh T. Vo, Jill M. Van Dalfsen, Sachinkumar B.P. Singh, Wendy Ni, Lucas R. Hoffman, Kailee McGeer, Sonya L. Heltshe, John P. Clancy, Steven M. Rowe, Peter Jorth, Pradeep K. Singh, the PROMISE-Micro Study Group   +14 more
doaj   +1 more source

Assessing Risk and Return: Personalized Medicine Development & New Innovation Paradigm [PDF]

, 2008
Outlines how focusing on subpopulations with biomarkers could make the pre-clinical and clinical phases of drug innovation more efficient and create economic returns.
Frank L. Douglas, Lesa Mitchell
core   +1 more source

The Race to Salvage Glucocerebrosidase: Understanding Small‐Molecule Therapies for GBA1‐Associated Parkinsonism

Movement Disorders, EarlyView.
Variants in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, cause Gaucher disease and confer an increased risk for parkinsonism. Strategies using small molecules can improve the function of glucocerebrosidase in lysosomes. A clear understanding of the mechanism‐of‐action of these compounds will facilitate development of GBA1‐modulating
Mark J. Henderson, Tiffany C. Chen, Logan M. Glasstetter, Yu Chen, Juan J. Marugan, Ellen Sidransky   +5 more
wiley   +1 more source

CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids

Orphanet Journal of Rare Diseases
Background Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio   +6 more
doaj   +1 more source

Attenuation of Phosphorylation-dependent activation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) by disease-causing mutations at the transmission interface [PDF]

, 2016
Cystic fibrosis transmembrane conductance regulator (CFTR) is a multidomain membrane protein that functions as a phosphorylation-regulated anion channel. The interface between its two cytosolic nucleotide binding domains and coupling helices conferred by
Bear, C.E., Chin, S., Eckford, P.D.W., Miles, Andrew J., Molinski, S., Wallace, Bonnie A., Yang, D.   +6 more
core   +1 more source

Interrelationships among handgrip strength, body composition, physical activity, and quality of life in adults with cystic fibrosis: A cross‐sectional study

Nutrition in Clinical Practice, EarlyView.
Abstract Background In individuals with cystic fibrosis (CF), lean mass and muscle strength are important predictors of clinical outcomes. This study evaluated associations among body composition, handgrip strength, muscle quality, physical activity, and health‐related quality of life in CF. Methods This observational, cross‐sectional study included 27
Benjamin H. Crain, Carly Harris, Moriah P. Bellissimo, Lucia A. Gonzalez Ramirez, Elizabeth A. Ivie, William R. Hunt, Vin Tangpricha, Thomas R. Ziegler, Ryan A. Harris, Jessica A. Alvarez   +9 more
wiley   +1 more source