Results 41 to 50 of about 21,301 (157)
IntroductionMutations affecting the RAS-MAPK pathway occur frequently in relapsed neuroblastoma tumors and are associated with response to MEK inhibition in vitro.
Thomas F. Eleveld +19 more
doaj +1 more source
Regulatory harmonization: Evolution, globalization and future directions
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 +2 more
wiley +1 more source
Aim Increasing use of oral antitumour therapeutics (OAT) in paediatrics is associated with numerous advantages, but also with error‐prone aspects. Based on the randomized AMBORA trial including adults treated with various OAT, we developed a tailored pharmacological/pharmaceutical care programme for children and their caregivers during OAT treatment ...
Phyllis Lensker +4 more
wiley +1 more source
Trametinib Sensitivity is Defined by a Myeloid Differentiation Profile in Acute Myeloid Leukemia
Background and Objective Acute myelogenous leukemia (AML) is a common blood cancer marked by heterogeneity in disease and diverse genetic abnormalities. Additional therapies are needed as the 5-year survival remains below 30%.
Mathieu Quesnel-Vallières +10 more
doaj +1 more source
Precision therapies for genetic epilepsies in 2025: Promises and pitfalls
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 +3 more
wiley +1 more source
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 +6 more
doaj +1 more source
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 +7 more
doaj +1 more source
ABSTRACT Biliary tract cancers (BTCs) are aggressive malignancies associated with a poor prognosis. Although molecular profiling is recommended to guide therapeutic decision‐making, real‐practice data on the prevalence and prognostic significance of genomic alterations in European BTC cohorts remain limited.
Jorine Arnouts +14 more
wiley +1 more source
Cutaneous Rosai‐Dorfman Disease With MAP2K1 Mutation Treated With Encorafenib and Binimetinib
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 +2 more
wiley +1 more source
MEK Inhibitor Associated Airway Injury in an Infant With Noonan Syndrome: A Case Report
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 +4 more
wiley +1 more source

