Results 211 to 220 of about 72,813 (290)

Long‐term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real‐world cohort study

British Journal of Clinical Pharmacology, EarlyView.
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta, Luigi Angelo Fiorito, Roberta Vescovo, Anna Virgilio, Giulia Amato, Giuseppe Cimino   +5 more
wiley   +1 more source

Population Physiologically-Based Pharmacokinetic Modeling to Determine Ontogeny: A Quantitative Clinical Pharmacology Example in Pediatric Rare Disease. [PDF]

CPT Pharmacometrics Syst Pharmacol
Cleary Y, Prasad B, Ogungbenro K, Gertz M, Galetin A.   +4 more
europepmc   +1 more source

Beyond the label: Rethinking off‐label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy

British Journal of Clinical Pharmacology, EarlyView.
Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden, Miriam G. Mooij, Matthijs de Hoog, Saskia N. de Wildt   +3 more
wiley   +1 more source

Solute Carrier transporters in tumor metabolism and immune modulation: implications for therapy. [PDF]

J Transl Med
Zhou J, Wen W, Xu Y, Yu J, Chen D.
europepmc   +1 more source

Efficacy, safety and cost‐effectiveness of CAR‐T therapy

British Journal of Clinical Pharmacology, EarlyView.
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher, Amina Džidić‐Krivić, Emma Pinjic, Nejra Selak, Kanita Omerbasic, Alexandar Chupin, Andrej Belancic, Almir Fajkic   +7 more
wiley   +1 more source

Role of solute carrier transporters in the biodistribution and toxicity of chemotherapeutic drugs. [PDF]

Pharmacol Rev
Boeckman M, Drabison T, Germakovski A, Warmuth A, Ahmed B, Chowdhury AT, Hu S, Sprowl JA, Sparreboom A, Huang KM.   +9 more
europepmc   +1 more source

The quantitative impact of metabolism‐inhibiting drugs on the occurrence of adverse drug reactions—A backward selection approach

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim The quantitative effect of several inhibitory drugs on the development of adverse drug reactions (ADRs) is currently difficult to estimate. Our aim was to identify metabolic pathways, which, when inhibited, increase the risk for certain ADRs, and to use this system to consider comedication at individual level. Methods Data of a prospective
Judith Berres, Justyna Wozniak, Hannah Fölsch, Anja Knueppel‐Ruppert, Verena Graeff, Andrea Kriegisch‐Stumpf, Harald Dormann, Julia C. Stingl, Katja S. Just   +8 more
wiley   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Guiling prescription attenuates hyperuricemia via multi-target regulation of uric acid metabolism, renal protection, and inflammation: insights from metabolomics and network pharmacology. [PDF]

Front Nutr
Wang Y, Ding R, Guo Y, Zhou T, Zhao H, Liu H, Qin X, Gao X.   +7 more
europepmc   +1 more source