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The PROPHECI trial: a phase II, double-blind, placebo-controlled, randomized clinical trial for the treatment of pseudoxanthoma elasticum with oral pyrophosphate.
Trials
January 2025
Université Côte d'Azur, CNRS, LP2M, Nice, France.
Background: /aims. Pseudoxanthoma Elasticum (PXE, OMIM 264800) is an autosomal, recessive, metabolic disorder characterized by progressive ectopic calcification in the skin, the vasculature and Bruch's membrane. Variants in the ABCC6 gene are associated with low plasma pyrophosphate (PPi) concentration.
View Article and Find Full Text PDFThe impact of universal mental health screening on stigma in primary schools.
Child Adolesc Psychiatry Ment Health
January 2025
Black Dog Institute, University of New South Wales, Sydney, NSW, Australia.
Background: Primary school students struggling with mental health are less likely than high school students to access mental health care, due to barriers such as mental health stigma and low mental health literacy among children and parents. The near universal reach of schools offers a potential avenue to increase access to mental health care through early identification. The potential risks of this approach also need to be understood.
View Article and Find Full Text PDFValidating a clinically based MS-MLPA threshold through comparison with Sanger sequencing in glioblastoma patients.
Clin Epigenetics
January 2025
Center of Oncocytogenomics, Institute of Medical Biochemistry and Laboratory Diagnostics, General University Hospital and 1st Faculty of Medicine of Charles University in Prague, U Nemocnice 499/2, 128 00, Prague, Czech Republic.
Background: Glioblastoma is the commonest malignant brain tumor and has a very poor prognosis. Reduced expression of the MGMT gene (10q26.3), influenced primarily by the methylation of two differentially methylated regions (DMR1 and DMR2), is associated with a good response to temozolomide treatment.
View Article and Find Full Text PDFSimulation-based training program effect on pediatric nurses' knowledge and performance regarding heel-prick during newborn blood screening test.
BMC Nurs
January 2025
Faculty of Applied Medical Sciences in Alnamas, University of Bisha, 255, Al Nakhil, Al-Namas, 67714, Saudi Arabia.
Background: One of the best ways to impart important skills to trainees is through simulation-based training, which is more reliable than the conventional clinical examination method. It is used in pediatric nursing training to allow nurses to practice and improve their clinical and conversational skills during an actual child encounter. A heel-prick is a complex psychomotor task that requires skill and knowledge from the pediatric nurse performing the procedure while applying for the National Newborn Screening Program.
View Article and Find Full Text PDFTAD-SIE: sample size estimation for clinical randomized controlled trials using a Trend-Adaptive Design with a Synthetic-Intervention-Based Estimator.
Trials
January 2025
Department of Electrical and Computer Engineering, Princeton University, Princeton, 08544, NJ, USA.
Background: Phase-3 clinical trials provide the highest level of evidence on drug safety and effectiveness needed for market approval by implementing large randomized controlled trials (RCTs). However, 30-40% of these trials fail mainly because such studies have inadequate sample sizes, stemming from the inability to obtain accurate initial estimates of average treatment effect parameters.
Methods: To remove this obstacle from the drug development cycle, we present a new algorithm called Trend-Adaptive Design with a Synthetic-Intervention-Based Estimator (TAD-SIE) that powers a parallel-group trial, a standard RCT design, by leveraging a state-of-the-art hypothesis testing strategy and a novel trend-adaptive design (TAD).
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