Results 31 to 40 of about 1,154,769 (315)

Personalized Selumetinib Dosing in Pediatric Neurofibromatosis Type 1: Insights From a Pilot Therapeutic Drug Monitoring Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Objective To evaluate selumetinib exposure using therapeutic drug monitoring (TDM) in pediatric patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN), assess interpatient pharmacokinetic variability, and explore the relationship between drug exposure, clinical response, and adverse effects.
Janka Kovács, Zoltán Köllő, Balázs Gellért Karvaly, Mahmoud Obeidat, Rita Nagy, Péter Hegyi, Brigitta Teutsch, Eszter Tuboly, Miklós Garami   +8 more
wiley   +1 more source

Adeno-associated virus delivered CXCL9 sensitizes glioblastoma to anti-PD-1 immune checkpoint blockade

Nature Communications
There are numerous mechanisms by which glioblastoma cells evade immunological detection, underscoring the need for strategic combinatorial treatments to achieve appreciable therapeutic effects.
Christina A. von Roemeling, Jeet A. Patel, Savannah L. Carpenter, Oleg Yegorov, Changlin Yang, Alisha Bhatia, Bently P. Doonan, Rylynn Russell, Vrunda S. Trivedi, Kelena Klippel, Daniel H. Ryu, Adam Grippin, Hunter S. Futch, Yong Ran, Lan B. Hoang-Minh, Frances L. Weidert, Todd E. Golde, Duane A. Mitchell   +17 more
doaj   +1 more source

Expanded specific T cells to hypomutated regions of the SARS-CoV-2 using mRNA electroporated antigen-presenting cells

Molecular Therapy: Methods & Clinical Development
The COVID-19 pandemic has caused about seven million deaths worldwide. Preventative vaccines have been developed including Spike gp mRNA-based vaccines that provide protection to immunocompetent patients. However, patients with primary immunodeficiencies,
Elizabeth Ogando-Rivas, Paul Castillo, Changlin Yang, Vrunda Trivedi, Dingpeng Zhang, Fernanda Pohl-Guimarães, Ruixuan Liu, Arnav Barpujari, Kate M. Candelario, Hector Mendez-Gomez, Elias J. Sayour, Duane A. Mitchell   +11 more
doaj   +1 more source

Adherence to Protocol Recommendations for Children With Wilms Tumour in Two Consecutive Studies in the United Kingdom and Ireland—Does Variation Matter?

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background and Aims Wilms tumour (WT) has excellent event‐free and overall survival (OS). However, small differences exist between countries participating in the same international study. This led us to examine variation in adherence to protocol recommendations as a potential contributing factor.
Suzanne Tugnait, R. Al‐Saadi, A. Lopez‐Cortes, K. Dzhuma, M. Oostveen, J. Brok, S. Irtan, M. Weeks, J. Bate, S. Baker, R. Williams, M. Powis, D. Saunders, O. Olsen, T. Watson, S. Shelmerdine, S. Vaidya, L. Howell, C. Duncan, G. M. Vujanic, C. Stiller, G. P. Balchin, T. Chowdhury, K. Pritchard‐Jones   +23 more
wiley   +1 more source

Patient Perspectives on Late-Onset Pompe Disease: Insights From a 2025 Patient Snapshot Survey on Diagnosis, Treatment, and Quality of Life

Journal of Patient Experience
Late-onset Pompe disease (LOPD) is a rare neuromuscular disorder, where delays in diagnosis can result in inconsistent patient management and suboptimal use of effective treatments, thereby increasing patient burden.
Benjamin W. Gallarda PhD, Ross A. Leslie MS, Felicia Oyedepo MD, Claire Almeida MS, Kevin Ampeh MPH, Guiomar Gonzalez BS, Benedikt Schoser MD   +6 more
doaj   +1 more source

All's Well That Ends Well, or Is It?

Journal of the American College of Cardiology, 2010
The past 2 decades have witnessed an explosion of basic and clinical research in atrial fibrillation (AF), and the pharmacologic and nonpharmacologic therapeutic options have improved tremendously ([1][1]).
Eric N, Prystowsky, Benzy J, Padanilam
openaire   +2 more sources

Sirolimus for Extracranial Arteriovenous Malformations: A Scoping Review of the Evidence in Syndromic and Non‐Syndromic Cases

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Arteriovenous malformations (AVMs) are rare, high‐flow, vascular anomalies that can occur either sporadically or as part of a genetic syndrome. AVMs can progress with serious morbidity and even mortality if left unchecked. Sirolimus is an mTOR inhibitor that is effective in low‐flow vascular malformations; however, its role in AVMs is unclear.
Will Swansson, Ingrid Winship, Gary Hammerschlag, Laura Scardamaglia   +3 more
wiley   +1 more source

Description of steady inflow of fluid to wells with different configurations and various partial drilling-in

Записки Горного института, 2020
There are many equations of steady inflow of fluid to the wells depending on the type of well, presence or absence of artificial or natural fractures passing through the well, different degrees of drilling-in of the wellbores.
Valery A. Iktissanov
doaj   +1 more source

Evaluating the Utility of Paired Tumor and Germline Targeted DNA Sequencing for Pediatric Oncology Patients: A Single Institution Report

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Objective To evaluate the diagnostic yield and utility of universal paired tumor–normal multigene panel sequencing in newly diagnosed pediatric solid and central nervous system (CNS) tumor patients and to compare the detection of germline pathogenic/likely pathogenic variants (PV/LPVs) against established clinical referral criteria for cancer ...
Natalie Waligorski, Rebecca Ronsley, Shannon M. Stasi, Jeffrey Stevens, Erin R. Rudzinski, Christina M. Lockwood, Sarah E. S. Leary, Bonnie L. Cole, Michelle A. Ting, Vera Paulson   +9 more
wiley   +1 more source

Drinking water quality and public health in Southwestern Saudi Arabia: The need for a national monitoring program

Journal of Family and Community Medicine, 2015
Aim of the Study: The aim was to investigate the bacteriological quality of drinking water, and explore the factors involved in the knowledge of the public about the quality of drinking water in Najran region, Saudi Arabia.
Jobran M Alqahtani, Ahmed M Asaad, Essam M Ahmed, Mohamed A Qureshi   +3 more
doaj   +1 more source