Results 161 to 170 of about 1,481,989 (336)

Population pharmacokinetics and dose–response relationships of mitoxantrone in children with acute myeloid leukaemia

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Background Information on mitoxantrone pharmacokinetics in children is lacking and reduced dosing regimens applied to infants are supported by limited scientific rationale. The current study characterized mitoxantrone pharmacokinetics in a childhood acute myeloid leukaemia patient population and provides a data‐informed assessment of dosing.
Andrew M. Brandon   +13 more
wiley   +1 more source

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

open access: yesBritish 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   +3 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

open access: yesBritish 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   +7 more
wiley   +1 more source

Opportunity analysis of newborn screening programs [PDF]

open access: yes
There exist congenital diseases that reduce newborns' potential opportunities. This reduction is sometimes alleviated if the congenital disease is early detected thanks to a newborn screening program.
HERRERO, Carmen, MORENO-TERNERO, Juan D.
core  

The decline of 6‐thioguanine nucleotides is not linked to impaired efficacy or safety of thiopurines in pregnant women with inflammatory bowel disease

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Background Thiopurines are used to maintain remission in inflammatory bowel disease (IBD). These drugs are metabolized into 6‐thioguanine nucleotides (6‐TGN), associated with efficacy, and 6‐methylmercaptopurine ribonucleotides (6‐MMPR), associated with adverse drug reactions. Pregnancy has been linked to a shift in thiopurine metabolism, characterized
Dianne G. Bouwknegt   +13 more
wiley   +1 more source

Randomized, placebo-controlled phase 3 trial evaluating safety, immunogenicity, and reactogenicity of RSVPreF3-Mat in high-risk pregnant women and their infants. [PDF]

open access: yesHum Vaccin Immunother
Bebia Z   +14 more
europepmc   +1 more source

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