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Benchmarking is an important step in the improvement, assessment, and comparison of the performance of drug discovery platforms and technologies. We revised the existing benchmarking protocols in our Computational Analysis of Novel Drug Opportunities (CANDO) multiscale therapeutic discovery platform to improve utility and performance. We optimized multiple parameters used in drug candidate prediction and assessment with these updated benchmarking protocols.
View Article and Find Full Text PDFTwo-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients.
Sci Rep
January 2025
Division of Metabolic Disorders, CHOC Children's Hospital, Orange, CA, 92868, USA.
Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rapidly progressive neurodegenerative disorder leading to premature mortality. Ambulatory CLN2 patients typically receive standard of care treatment through biweekly intracerebroventricular (ICV) enzyme replacement therapy (ERT) involving recombinant human tripeptidyl peptidase 1, known as cerliponase alfa (Brineura, Biomarin Pharmaceuticals). This study longitudinally assessed the impact of ICV cerliponase alfa ERT on gait, and postural control across a two-year span in two siblings diagnosed with atypical CLN2 disease.
View Article and Find Full Text PDFImproving the evaluation of non-accidental trauma across multiple specialties at a single institution.
BMJ Open Qual
January 2025
Inova Fairfax Medical Campus, Falls Church, Virginia, USA.
Background: Based on the presenting injury, patients undergoing abuse evaluation may be managed by different specialties. Our local child abuse specialist expressed concern over the variability in evaluation of patients presenting with injuries concerning for non-accidental trauma (NAT). The aim of this quality improvement project was to increase the percentage of patients for whom there is a concern for NAT who receive a guideline-adherent evaluation from 7.
View Article and Find Full Text PDFAssessing the Risk for Falls in Older Adults After Initiating Gabapentin Versus Duloxetine.
Ann Intern Med
January 2025
Center for Healthcare Delivery Sciences, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts (R.J.D., N.K.C., N.H., J.C.L.).
Background: The evidence informing the harms of gabapentin use are at risk of bias from comparing users with nonusers.
Objective: To describe the risk for fall-related outcomes in older adults starting treatment with gabapentin versus duloxetine.
Design: New user, active comparator study using a target trial emulation framework.
Effects of metformin on postprandial blood pressure, heart rate, gastric emptying, GLP-1 and the prevalence of postprandial hypotension in type 2 diabetes - a double-blind, placebo-controlled crossover study.
Diabetes
January 2025
Adelaide Medical School and Centre of Research Excellence (CRE) in Translating Nutritional Science to Good Health, The University of Adelaide, Adelaide, Australia.
Individuals with type 2 diabetes are at high risk of postprandial falls in blood pressure (BP) (i.e., a reduction in systolic BP of ≥20mmHg, termed postprandial hypotension (PPH)), which increases the risk of falls and mortality.
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