Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients.

A new version of Fisher’s discriminant analysis (FDA) is introduced in this paper. Our algorithm searches also for a reduced space in which patterns can be discriminated. However, no intermediate class separability criterion (such as Fisher’s mean distance divided by variance) is used whatsoever. Classification performance is optimized directly. Since no statistical hypothesis are made, the met...

Some individual fish like to be close together in 'schools', while other individuals like to be alone. A pair of recent papers dissects the genetic basis of schooling behavior, showing that genetic changes in sensory systems are involved when this social behavior is lost during evolution.

Efficient and stoichiometric expression of genes concatenated by bi- or multi-cistronic vectors has become an invaluable tool not only in basic biology to track and visualize proteins in vivo, but also for vaccine development and in the clinics for gene therapy. To adequately compare, in vivo, the effectiveness of two of the currently popular co-expression strategies - the internal ribosome ent...

This work aims to measure the impact of approximate computing on a case study traffic dynamics metering System-on-Chip edge device. Firstly, proposes baseline implementation system in C++. To analyze application detail, profiled using built-in instrumented profiler, presenting overall performance each its parts. During hotspot analysis, some parts had optimization opportunities exploitable by m...

BACKGROUND Nonadherence among hemodialysis patients compromises dialysis delivery, which could influence patient morbidity and mortality. The Dialysis Outcomes and Practice Patterns Study (DOPPS) provides a unique opportunity to review this problem and its determinants on a global level. METHODS Nonadherence was studied using data from the DOPPS, an international, observational, prospective h...

Exon skipping is considered a principal mechanism by which eukaryotic cells expand their transcriptome and proteome repertoires, creating different splice variants with distinct cellular functions. Here we analyze RNA-seq data from 116 transcriptomes in fission yeast (Schizosaccharomyces pombe), covering multiple physiological conditions as well as transcriptional and RNA processing mutants. We...

Aktivitas fisik berupa olahraga lari, jalan cepat, skipping dengan intesitas sedang dapat meningkatkan beberapa fungsi kognitif salah satunya adalah memori. Memori jangka pendek memliki peran yang sangat penting dalam kehidupan terutama proses pengelolahan informasi. Penelitian ini bertujuan untuk membuktikan pengaruh aktivitas dilakukan secara rutin memori pendek. Metode penelitian digunakan k...

High speed trains are an indispensable part of rapid transportation systems in many countries. As the fastest means of transport on the ground, these trains are extremely sensitive to the terrain and track conditions. Geometrical irregularities and mechanical characteristics of the track, together with inconsistencies in the lane (including bridges that support the track) are considered importa...