Maximizing the impact of megaprojects: Urgent implications of financial inclusion drive for effective anti-poverty measures.

Megaprojects are becoming more and more essential for poverty reduction and inclusive development. Regrettably, the CPEC mega project has yet to yield successful outcomes in Pakistan. The incorporation of financial inclusion (FI) has the potential to catalyze its outcomes.

Rank and Wormhole Attack Detection Model for RPL-Based Internet of Things Using Machine Learning.

The proliferation of the internet of things (IoT) technology has led to numerous challenges in various life domains, such as healthcare, smart systems, and mission-critical applications. The most critical issue is the security of IoT nodes, networks, and infrastructures. IoT uses the routing protocol for low-power and lossy networks (RPL) for data communication among the devices.

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized, double-blind, placebo-controlled, dose-titration study followed by 9-week safety review. Part 2 was a 168-week, open-label evaluation of golodirsen 30 mg/kg.

5G and IoT Based Reporting and Accident Detection (RAD) System to Deliver First Aid Box Using Unmanned Aerial Vehicle.

Internet of Things (IoT) and 5G are enabling intelligent transportation systems (ITSs). ITSs promise to improve road safety in smart cities. Therefore, ITSs are gaining earnest devotion in the industry as well as in academics.

Prevalence and impact of the use of electronic gadgets on the health of children in secondary schools in Bangladesh: A cross-sectional study.

Background And Aims: Use of technological gadgets has rapidly been increasing among adolescents, which may result in health issues and technology addiction. This study focuses on the prevalence of usage of technological gadgets and health-related complications among secondary school-going children of Bangladesh.

Methods: A total of 1803 secondary school students from 21 different districts of Bangladesh participated in the study.

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production.ObjectiveTo report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficacy and safety of eteplirsen in a larger cohort.MethodsAmbulatory patients aged 7-16 years, with confirmed mutations amenable to exon 51 skipping, received eteplirsen 30 mg/kg/week intravenously for 96 weeks.

Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.

Background: Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by DMD gene mutations. A relationship between exon skipping and dystrophin production in exon 51-amenable patients treated with eteplirsen (EXONDYS 51®) is established. Once-weekly eteplirsen significantly increased dystrophin, with slower decline in ambulatory function compared to baseline.

Clinical development on the frontier: gene therapy for duchenne muscular dystrophy.

: The development of adeno-associated virus (AAV) vectors as safe vehicles for delivery of therapeutic genes has been a major milestone in the advancement of gene therapy, enabling a promising strategy for ameliorating a wide range of diseases, including Duchenne muscular dystrophy (DMD).: Based on experience with the development of a gene transfer therapy agent for DMD, we discuss ways in which gene therapy for rare disease challenges traditional clinical development paradigms, and recommend a step-wise approach for design and evaluation to support broader applicability of gene therapy.: The gene therapy development approach should intentionally design the therapeutic construct and the clinical study to systematically evaluate agent delivery, safety, and efficacy.

Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy.

Background: Duchenne muscular dystrophy (DMD) patients experience skeletal muscle degeneration, including respiratory muscles. Respiratory decline in glucocorticoid-treated DMD patients, measured by percent predicted forced vital capacity (FVC% p), is typically 5% annually in patients aged 10 to 18 years.

Objective: Evaluate the effects of eteplirsen on FVC% p annual change in 3 trials versus matched Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS) controls.

A novel primary amine-based anion exchange membrane adsorber.

A novel anion exchange membrane adsorber is presented which shows excellent impurity removal under different buffer conductivities ranging from 2 to 2 7mS/cm. The membrane utilizes a primary amine ligand (polyallylamine) and was designed specifically to bind impurities at high salt concentrations. Studies with DNA, endotoxin, and virus spiked into buffer at varying salt conditions were done, resulting in clearance of >3, 4, and 4 LRV, respectively, with negligible change on increasing salt up to 27 mS/cm conductivities.

Filter preconditioning enables representative scaled-down modelling of filter capacity and viral clearance by mitigating the impact of virus spike impurities.

Endogenous and adventitious virus removal by size-exclusion membrane filtration is a critical dedicated step in an overall viral clearance strategy employed by biologics manufacturers as required by industry regulators. However, the addition of impurities from virus spike preparations used in validation studies can significantly reduce filter capacity, resulting in an oversized and suboptimal virus filtration step. The hydraulic filter performance and virus retention observed in conventional scaled-downed validation models may not necessarily represent performance observed during process development, nor be predictive of manufacturing performance.