Results 41 to 50 of about 20,250 (180)

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Regulatory harmonization: Evolution, globalization and future directions

British Journal of Clinical Pharmacology, EarlyView.
Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm, Elaine Y. L. Blair, Christine Y. Lu   +2 more
wiley   +1 more source

Case Report: Trametinib in the treatment of patients with metastatic lung adenocarcinoma harboring NF1 mutation: a case series and literature review

Frontiers in Oncology
BackgroundThe neurofibromin 1 (NF1) protein regulates the downstream RAS/RAF/MEK/ERK pathway and functions as a tumor suppressor. Somatic pathogenic mutations in NF1 are found in approximately 4.7%–10% of NSCLC, with a higher frequency in lung ...
Floryane Kim, Maxime Borgeaud, Maxime Borgeaud, Claudio De Vito, Petros Tsantoulis, Petros Tsantoulis, Alfredo Addeo   +6 more
doaj   +1 more source

Inhibition of MEK with trametinib enhances the efficacy of anti-PD-L1 inhibitor by regulating anti-tumor immunity in head and neck squamous cell carcinoma

OncoImmunology, 2019
Major histocompatibility complex (MHC) class I downregulation is the primary immune evasion mechanism associated with failure in anti-PD-1/PD-L1 blockade therapies for cancer.
Seong-Ho Kang, Bhumsuk Keam, Yong-Oon Ahn, Ha-Ram Park, Miso Kim, Tae Min Kim, Dong-Wan Kim, Dae Seog Heo   +7 more
doaj   +1 more source

Early‐Phase Research in Pediatric Neuro‐Oncology

Pediatric Blood &Cancer, EarlyView.
Jonathan Cunningham
wiley   +1 more source

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Advances in Immunological Combination Therapy for Colorectal Cancer

iMetaMed, EarlyView.
Review the progress of combined immunotherapy for colorectal cancer. ABSTRACT Immune checkpoint inhibitors (ICIs) have been utilized extensively and shown to be therapeutically beneficial in the treatment of solid tumors in recent years. Nonetheless, considerable heterogeneity in Colorectal cancer (CRC) requires careful consideration.
Jiayue Wu, Rongbo Wen, Tianshuai Zhang, Fuao Cao, Xiaoming Zhu, Liqiang Hao, Wei Zhang, Guanyu Yu   +7 more
wiley   +1 more source

A Phase 1/1B Trial of Pembrolizumab and Trametinib in Advanced NSCLC Enriched for KRAS Mutations

JTO Clinical and Research Reports
Introduction: MEK inhibition (MEKi) combined with programmed death ligand 1 inhibition (immune checkpoint inhibitor [ICI]) modulates the tumor immune microenvironment.
Jonathan W. Riess, MD, MS, Matthew S. Lara, BS, Guillaume Luxardi, PhD, Miguel Lopez de Rodas, MD, Michiko Shimoda, PhD, Karen Kelly, MD, Primo N. Lara, MD, Laurel Beckett, PhD, Arta Monjazeb, MD, PhD, Kurt A. Schalper, MD, PhD, Emanual Maverakis, MD, David R. Gandara, MD   +11 more
doaj   +1 more source

Cutaneous Rosai‐Dorfman Disease With MAP2K1 Mutation Treated With Encorafenib and Binimetinib

JEADV Clinical Practice, EarlyView.
ABSTRACT Rosai‐Dorfman disease (RDD) is a rare histiocytic disorder with unifocal to multisystemic involvement. Activating mutations in the MAPK/ERK pathway can occur in up to 50% of RDD cases. We report a case of cutaneous RDD (C‐RDD) with facial and truncal lesions harboring an activating MAP2K1 (MEK1) mutation that showed a complete response to a ...
Melike Ak, Reinhard Dummer, Egle Ramelyte   +2 more
wiley   +1 more source

MEK Inhibitor Associated Airway Injury in an Infant With Noonan Syndrome: A Case Report

The Laryngoscope, EarlyView.
An infant with Noonan syndrome treated with trametinib developed extensive mucosal sloughing of the upper and lower airway, followed by severe supraglottic scarring. Clinicians should consider airway toxicity as a potential adverse effect of MEK inhibitor therapy.
Veronica Drozdowski‐Nuccio, Rachel D. Yoon, Steven Tomasi, Dana M. Thompson, Inbal Hazkani   +4 more
wiley   +1 more source