Gastroenterologically relevant high alert medications prescribed to children with chronic diseases-a consensus-driven single-center pilot study. [PDF]
Hochrainer J +4 more
europepmc +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Evaluating species at risk in data-limited fisheries: A productivity-susceptibility analysis for marine aquarium fish. [PDF]
Baillargeon GA +4 more
europepmc +1 more source
Uncovering G Protein‐Coupled Receptors: Novel Targets and Biomarkers for Predicting Glioma Prognosis
ABSTRACT Background Low‐grade gliomas (LGG) exhibit significant heterogeneity and recurrence risk. G protein‐coupled receptors (GPCR) contribute to glioma malignant progression, but their prognostic value remains unclear. This work attempts to formulate a GPCR‐based outcome‐predicting model for LGG. Methods Based on TCGA LGG data, the enrichment scores
Jun Yang +4 more
wiley +1 more source
The "China model" for rare disease governance: Policy framework, local implementation, and pathways for optimization. [PDF]
Zhang X +5 more
europepmc +1 more source
T1 Over Squared Proton Density Ratio to Characterize Multiple Sclerosis Lesions
ABSTRACT Objective Differentiating remyelinated from demyelinated lesions in MS remains challenging without histological confirmation. This study introduces the T1‐to‐PD2 ratio (TPR) imaging approach and evaluates its ability to characterize MS lesions alongside other quantitative MRI (qMRI) metrics. Methods Thirty individuals with MS (mean age: 47.5 ±
Sarah J. Wright +10 more
wiley +1 more source
Accelerated long-term forgetting as a predictor of clinical onset in presymptomatic autosomal dominant Alzheimer's disease. [PDF]
Magill N +9 more
europepmc +1 more source
ABSTRACT Objectives Focal cortical dysplasia (FCD) is the most common etiology of drug‐resistant epilepsy in children. Focal to bilateral tonic–clonic seizures (FBTCS) mark a high risk of drug‐resistant epilepsy and involve thalamocortical circuitry in their generation and propagation.
Hua Xie +8 more
wiley +1 more source
Correction: Emerging and re-emerging vector-borne and other zoonotic RNA viruses: pathogenesis, climate-driven dynamics, and strategies for global control. [PDF]
Frontiers Production Office.
europepmc +1 more source
Portal Vein Thrombosis in Cirrhotic Candidates for Liver Transplantation and Its Impact on the Transplant Accessibility. [PDF]
Parisse S +9 more
europepmc +1 more source

