Results 151 to 160 of about 1,105,613 (399)

Discovery of the Principal Cystic Fibrosis Mutation (F508del) in Ancient DNA from Iron Age Europeans [PDF]

open access: yes, 2007
The most common, life-threatening autosomal recessive disease of Europeans and Euro-Americans, cystic fibrosis (CF), occurs predominately in patients with the F508del mutation.1 Although F508del is currently detectable as a single allele in 1/30-1/40 ...
Cedric Le Marechal   +4 more
core   +1 more source

Development of Novel Anticancer Pyrazolopyrimidinones Targeting Glioblastoma

open access: yesChemMedChem, EarlyView.
Pyrazolo[1,5‐α]pyrimidinone derivatives exhibit selective cytotoxicity towards glioblastoma (GBM) cells over noncancerous cells. Structure–activity relationship studies identifies a lead compound with significant cytotoxicity activity, inducing apoptosis and necrosis in GBM cells, without affecting noncancerous cells. This demonstrates the potential of
Kate Byrne   +5 more
wiley   +1 more source

Recent Insights in Multi‐Target Drugs in Pharmacology and Medicinal Chemistry

open access: yesChemMedChem, EarlyView.
This review highlights the rationale behind multitarget drug design as a promising approach to address diseases with complex etiologies. By combining pharmacophore features from different single‐target drugs, multitarget compounds can interact with multiple biological targets simultaneously.
Sadık Hüseyin Cemali   +7 more
wiley   +1 more source

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

open access: yesOrphanet 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   +6 more
doaj   +1 more source

Considerations in Transition of Pediatric Neurology Patients to Adult Care

open access: yesAnnals of the Child Neurology Society, EarlyView.
ABSTRACT Background Transition refers to the planned, coordinated movement of adolescents from the child‐ and family‐centered environment of pediatric care to the adult healthcare system. A well‐structured transition process is essential for ensuring adolescents with chronic health conditions continue to thrive in young adulthood.
Asif Doja   +5 more
wiley   +1 more source

Detection of cytokines in nasal lavage samples of patients with cystic fibrosis: comparison of two different cytokine detection assays

open access: yesBMC Pulmonary Medicine
Background Cystic fibrosis (CF) is a genetic multisystem disorder. Inflammatory processes, which presumably begin early in infancy, play a crucial role in the progression of the disease. The detection of inflammatory biomarkers, especially in the airways,
Teresa Fuchs   +8 more
doaj   +1 more source

The future of cystic fibrosis care: a global perspective.

open access: yesThe Lancet Respiratory Medicine, 2020
S. Bell   +37 more
semanticscholar   +1 more source

Population Pharmacokinetic/Toxicodynamic Model for Polymyxin B in Critically Ill Patients to Identify the Risk of Nephrotoxicity

open access: yesClinical Pharmacology &Therapeutics, EarlyView.
Polymyxins are used against highly resistant Gram‐negative pathogens, and nephrotoxicity (acute kidney injury, AKI) is the major dose‐limiting adverse effect. We aimed to develop a well‐informed population pharmacokinetic/toxicodynamic (PK/TD) model using the largest polymyxin B‐treated critically ill patient population studied to inform polymyxin B ...
Patrick O. Hanafin   +19 more
wiley   +1 more source

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