Fenfluramine treatment for Dravet syndrome: Long term real-world analysis demonstrates safety and reduced health care burden.

Objective: Fenfluramine (FFA), stiripentol (STP), and cannabidiol (CBD) are approved add-on therapies for seizures in Dravet syndrome (DS). We report on the long-term safety and health care resource utilization (HCRU) of patients with DS treated with FFA under an expanded access program (EAP).

Methods: A cohort of 124 patients received FFA for a median of 2.

Clinical and genetic drivers of oligo-metastatic disease in colon cancer.

Background: Oligo-metastatic disease (OMD) in colon cancer patients exhibits distinct clinical behavior compared to poly-metastatic disease (PMD), with a more responsive and indolent course. This study aims to identify clinical and biological factors uniquely associated with oligo-metastatic behavior.

Methods: Metastatic colon cancer patients from an academic center underwent genetic characterization.

Convolutional neural networks for automatic MR classification of myocardial iron overload in thalassemia major patients.

Objectives: To develop a deep-learning model for supervised classification of myocardial iron overload (MIO) from magnitude T2* multi-echo MR images.

Materials And Methods: Eight hundred twenty-three cardiac magnitude T2* multi-slice, multi-echo MR images from 496 thalassemia major patients (285 females, 57%), labeled for MIO level (normal: T2* > 20 ms, moderate: 10 ≤ T2* ≤ 20 ms, severe: T2* < 10 ms), were retrospectively studied. Two 2D convolutional neural networks (CNN) developed for multi-slice (MS-HippoNet) and single-slice (SS-HippoNet) analysis were trained using 5-fold cross-validation.

High tumor mutational burden assessed through next-generation sequencing predicts favorable survival in microsatellite stable metastatic colon cancer patients.

Background: Microsatellite instability (MSI) is a well-established predictive biomarker for immune checkpoint inhibitor (ICI) response in metastatic colon cancer. Both high MSI and tumor mutational burden (TMB) are markers of genomic instability. However, the prognostic and predictive value of TMB in patients with microsatellite stable (MSS) tumors remains unclear.

Flexible 3D nanofiber-based SERS biosensor for detection of miRNA-223-3p in early Laryngeal Cancer diagnosis.

MicroRNAs (miRNAs) are small non-coding RNAs (18-22 nucleotides) that regulate gene expression and are associated with various diseases, including Laryngeal Cancer (LCa), which has a high mortality rate due to late diagnosis. Traditional methods for miRNA detection present several drawbacks (time-consuming steps, high cost and high false positive rate). Early-stage diagnosis and selective detection of miRNAs remain challenging.