Results 101 to 110 of about 104,915 (286)

Adverse immunostimulation in early phase clinical trials: Key findings and recommendations based on the investigator's clinical experience

British Journal of Clinical Pharmacology, EarlyView.
Problem setting The emergence of therapeutic proteins has coincided with an increase of acute adverse immunostimulation (AIS). AIS has occured in clinical trials despite compliance with regulatory guidelines on preclinical evaluation and its incidence is anticipated to increase even further.
Juliette A. van den Noort, Marella C. E. van Ruissen, Lisanne C. A. Smidt, Naomi B. Klarenbeek, Philip H. K. Kremer, Manon A. A. Jansen, Jacobus Burggraaf, Joop M. A. van Gerven, Matthijs Moerland   +8 more
wiley   +1 more source

Efficacy and safety of empagliflozin for treating neutropenia and neutrophil dysfunction in paediatric patients with glycogen storage disease type Ib: A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Aims Glycogen storage disease type Ib (GSD‐Ib) is a rare genetic disorder causing neutropenia and neutrophil dysfunction in children. G‐CSF has been the primary treatment, but emerging data support the potential of empagliflozin, an SGLT2 inhibitor, as a promising investigational option.
Elizabeth Iwasyk, Ryan Jin, Fabio Tuzzolino, Giusy Ranucci, Donatella Madonia, Alessio Provenzani   +5 more
wiley   +1 more source

Investigational and Experimental Drugs for Community-Acquired Pneumonia: the Current Evidence

Journal of Experimental Pharmacology, 2020
Juilia Sellarès-Nadal, Joaquin Burgos, Vicenç Falcó, Benito Almirante Infectious Diseases Department, Hospital Universitari Vall d’Hebron, Autonomous University of Barcelona, Barcelona, SpainCorrespondence: Benito ...
Sellarès-Nadal J, Burgos J, Falcó V, Almirante B   +3 more
doaj  

Mechanism‐informed machine learning for individualized tacrolimus dose adjustment in the early post‐kidney transplant period

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Tacrolimus dosing in the early post‐kidney transplant period is challenging due to a narrow therapeutic index and substantial interindividual pharmacokinetic (PK) variability. This study aimed to develop and validate mechanism‐informed machine learning (ML) models to support individualized tacrolimus dosing during this critical period ...
Hui Yu, Zihan Qin, Logan S. Smith, Jeong M. Park, Hao‐Jie Zhu   +4 more
wiley   +1 more source

Alzheimer's Disease: Novel Targets and Investigational Drugs for Disease Modification. [PDF]

Drugs, 2023
Cummings JL, Osse AML, Kinney JW.
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

Investigational Drugs for the Treatment of Postherpetic Neuralgia: Systematic Review of Randomized Controlled Trials. [PDF]

Int J Mol Sci, 2023
Huerta MÁ, Garcia MM, García-Parra B, Serrano-Afonso A, Paniagua N.   +4 more
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

Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs [PDF]

, 2018
While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment.
Bannister, Julien B.
core   +1 more source

Effect of carbamazepine on the pharmacokinetics of vepdegestrant, a PROteolysis TArgeting Chimera estrogen receptor degrader, in healthy adults

British Journal of Clinical Pharmacology, EarlyView.
Aim To evaluate the effects of carbamazepine, a strong cytochrome P450 (CYP)3A4 inducer, on the pharmacokinetics and safety of vepdegestrant, a PROteolysis TArgeting Chimera estrogen receptor degrader. Methods This was a phase 1, open‐label, fixed‐sequence, two‐period study in healthy adult participants.
Hechuan Wang, Jennifer A. Winton, Kyle T. Matschke, Alexandre Stouffs, Kimberly C. Lee, Yuanyuan Zhang, Wenlian Qiao, Weiwei Tan   +7 more
wiley   +1 more source