An improved SPWM control approach with aid of ant lion optimization for minimizing the THD in multilevel inverters.

This article presents an innovative asymmetric multilevel inverter (MLI) topology that outperforms conventional counterparts. The introduced topology presents a breakthrough in implementing power electronics control by maximizing specific levels while minimizing switching components. A cutting-edge control scheme for optimal operation of the cascaded half-bridge MLI is presented.

Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP).

Introduction: Gaucher disease type 3 (GD3) is a genetic, progressive lysosomal storage disorder characterized by visceral manifestations and chronic neurologic symptoms (e.g., horizontal ophthalmoplegia/supranuclear gaze palsy, ataxia, dystonia).

The value of knowing: preferences for genetic testing to diagnose rare muscle diseases.

Background: Genetic testing can offer early diagnosis and subsequent treatment of rare neuromuscular diseases. Options for these tests could be improved by understanding the preferences of patients for the features of different genetic tests, especially features that increase information available to patients.

Methods: We developed an online discrete-choice experiment using key attributes of currently available tests for Pompe disease with six test attributes: number of rare muscle diseases tested for with corresponding probability of diagnosis, treatment availability, time from testing to results, inclusion of secondary findings, necessity of a muscle biopsy, and average time until final diagnosis if the first test is negative.

A grey wolf optimization-based modified SPWM control scheme for a three-phase half bridge cascaded multilevel inverter.

The Multilevel inverter (MLI) plays a pivotal role in Renewable Energy (RE) systems by offering a cost-effective and highly efficient solution for converting DC from Photovoltaic (PV) sources into AC at high voltages. In addition, an innovative technology holds immense significance as it not only enables the seamless integration of PV systems into the grid but also ensures optimal power generation, thereby contributing to the widespread adoption of RE and fostering a sustainable future. This paper presents a modified sinusoidal pulse width modulation (SPWM) control scheme for a three-phase half-bridge cascaded MLI-powered PV sources.

Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.

Background: Clinical trials for rare diseases often include multiple endpoints that capture the effects of treatment on different disease domains. In many rare diseases, the primary endpoint is not standardized across trials. The win ratio approach was designed to analyze multiple endpoints of interest in clinical trials and has mostly been applied in cardiovascular trials.

Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.

Background: The Phase 3 COMET trial (NCT02782741) comparing avalglucosidase alfa and alglucosidase alfa included health-related quality of life (HRQoL) assessments in treatment-naïve patients with late-onset Pompe disease (LOPD). Here, we further characterize results from disease-specific and general patient-reported outcome (PRO) measures.

Methods: Adults who participated in the COMET trial receiving avalglucosidase alfa or alglucosidase alfa (both 20 mg/kg biweekly) during the 49-week double-blind treatment period were included in the analysis.

Caffeine-folic acid-loaded-chitosan nanoparticles combined with methotrexate as a novel HepG2 immunotherapy targeting adenosine A2A receptor downstream cascade.

Background: Methotrexate (MTX) is a common chemotherapeutic drug that inhibits DNA synthesis and induces apoptosis. Treatment with MTX increased CD73 expression, which leads to higher levels of extracellular adenosine. Adenosine levels are also high in the tumor microenvironment through Cancer cells metabolism.

Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study.

Background And Objectives: The Pompe Disease Symptom Scale (PDSS) and Impact Scale (PDIS) were created to measure the severity of symptoms and functional limitations experienced by patients with late-onset Pompe disease (LOPD). The objectives of this analysis were to establish a scoring algorithm and to examine the reliability, validity, and responsiveness of the measures using data from the COMET clinical trial.

Methods: The COMET trial was a randomized, double-blind study comparing the efficacy and safety of avalglucosidase alfa and alglucosidase alfa in patients with LOPD aged 16-78 years at baseline.

A new method for ferroresonance suppression in an IEEE 33-bus distribution system integrated with multi distributed generation.

Although integrating a distributed generation (DG) into a distribution system (DS) has several benefits, it may be accompanied by some issues, such as ferroresonance. Therefore, ferroresonance investigations in an integrated DS with multi-DGs have been identified as a research gap. To this end, this paper presents a new method to mitigate ferroresonance in distribution networks, after which ferroresonance in an IEEE-33 bus radial DS integrated with multi-DGs was investigated.

Variation of caesarean section rates in Palestinian governmental hospitals.

Background: Globally, the increased use of cesarean sections has become prevalent in high-income and low and middle-income countries. In Palestine, the rate had risen from 20.3% in 2014 to 25.

Long versus short segment with intermediate screw fixation for burst fractures of thoracolumbar junction: radiological and clinical results.

Background: Long segment fixation has been frequently used to treat thoracolumbar burst fractures. In our study we want to compare the long and short segment with intermediate screw fixation of thoracolumbar junction burst fractures in relation to radiological and clinical outcomes.

Methods: We collected the data of 115 patients, with thoracolumbar junction (T11-L2) burst fracture A3 or A4, according to AO Classification.

National mapping of schistosomiasis, soil-transmitted helminthiasis and anaemia in Yemen: Towards better national control and elimination.

Background: Schistosomiasis (SCH) and soil-transmitted helminthiasis (STH) are known to be endemic in Yemen. However, the distribution of both diseases had not previously been assessed by a well-structured national mapping study covering all governorates. The main aim of this study was, therefore, to map the prevalence of SCH and STH in Yemen in order to better inform implementation of effective national control and elimination interventions.

Fabry Disease Patient-Reported Outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease.

Background: Fabry disease (FD) is a rare, genetic disease, that if untreated, progresses to irreversible and life-threatening renal, cardiac, and cerebrovascular events. FD symptoms impact daily functioning and quality of life, but no disease-specific measure of these symptoms has been psychometrically tested.

Methods: The Fabry Disease Patient-Reported Outcome (FD-PRO) consists of 19 items that measure neuropathic symptoms (pain, tingling, numbness and burning in upper/lower extremities), headache, abdominal pain, heat intolerance, swelling, tinnitus, fatigue, hearing/vision impairment, hypohidrosis (diminished sweating) and difficulty engaging in regular physical activities in the past 24 h.

Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective.

Background: Late-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients' daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform the development of new patient-reported outcome (PRO) tools.

Development of the Fabry Disease Patient-Reported Outcome (FD-PRO): a new instrument to measure the symptoms and impacts of Fabry Disease.

Background: The systematic collection of disease-specific symptoms and impacts on the lives of patients with Fabry Disease (FD) can offer unique insights into the patient experience, yet no disease-specific tool to measure FD symptoms exists. This study describes the development of the Fabry Disease Patient-Reported Outcome (FD-PRO).

Methods: A targeted literature search, interviews with key opinion leaders (KOLs), and concept elicitation (CE) interviews with patients identified the most frequent signs and symptoms associated with FD and their impact on daily life.

Agalsidase beta treatment slows estimated glomerular filtration rate loss in classic Fabry disease patients: results from an individual patient data meta-analysis.

Background: Fabry disease is a rare, X-linked genetic disorder that, if untreated in patients with the Classic phenotype, often progresses to end-stage kidney disease. This meta-analysis determined the effect of agalsidase beta on loss of estimated glomerular filtration rate (eGFR) in the Classic phenotype using an expansive evidence base of individual patient-level data.

Methods: The evidence base included four Sanofi-Genzyme studies and six studies from a systematic literature review.

Patient and caregiver perspectives on burden of disease manifestations in late-onset Tay-Sachs and Sandhoff diseases.

Background: The GM2 gangliosidoses (GM2), Tay-Sachs and Sandhoff diseases, are rare, autosomal recessive genetic disorders caused by mutations in the lysosomal enzyme β-hexosaminidase A (HEXA) or β-hexosaminidase B (HEXB) genes, respectively. A minority of patients have a late-onset form of disease that presents from late-childhood to adulthood and has a slowly progressive course with prolonged survival. Little research has been published documenting patient experiences with late-onset Tay-Sachs and Sandhoff diseases and how the disease impacts their daily lives and functioning.

Induction of Apoptosis by Nano-Synthesized Complexes of H2L and its Cu(II) Complex in Human Hepatocellular Carcinoma Cells.

Background: Chemotherapy is currently the most utilized treatment for cancer. Therapeutic potential of metal complexes in cancer therapy has attracted a lot of interest. The mechanisms of action of most organometallic complexes are poorly understood.

Mobility assessment using wearable technology in patients with late-onset Pompe disease.

Late-onset Pompe disease (LOPD) is a rare genetic disorder due to the absence or deficiency of acid alpha-glucosidase enzyme resulting in slowly progressing reduction of muscle strength, causing difficulties with mobility and respiration. Wearable technologies offer novel options to evaluate mobility in a real-world setting. LOPD patients self-reporting LOPD, ≥18 years, US residents, walking (with or without aid), and not on invasive ventilation were recruited for a 6- to 8-week wearable study via patient organizations.

Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis.

Background: Late-onset Pompe disease (LOPD) is a rare, metabolic disease primarily affecting the musculoskeletal and respiratory systems. Forced vital capacity (FVC) is commonly used to measure pulmonary function; however, associations between FVC and other LOPD outcomes remain unclear.

Methods: A systematic literature review was conducted on November 2015, updated September 2016 and supplemented with clinical trial data from the sponsor.

The range and nature of reproductive health research in the occupied Palestinian territory: a scoping review.

Background: In order to set research priorities for reproductive health in the occupied Palestinian territory, it is vital to know what current research has been done in the field of reproductive health. The purpose of this scoping review is to examine the range and nature of reproductive health research in the occupied Palestinian territory and to identify research gaps in the existing literature.

Methods: We searched four databases: EMBASE, PubMed, CINAHL, and Popline.

A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease.

Background: This study aimed to obtain UK societal-based utility values for health states related to treatment mode of administration using Gaucher disease as the background condition.

Methods: A review of relevant literature and expert clinical input informed the development of five health states characterising the impact of Gaucher disease and its management on patients' lives. A base-state characterising the "controlled disease" was developed as well as four subsequent health states which varied in description of the method (intravenous versus oral) and frequency of treatment administration.

PATIENT-CENTERED DECISION MAKING: LESSONS FROM MULTI-CRITERIA DECISION ANALYSIS FOR QUANTIFYING PATIENT PREFERENCES.

Objectives: Patient preferences should be a central consideration in healthcare decision making. However, stories of patients challenging regulatory and reimbursement decisions has led to questions on whether patient voices are being considered sufficiently during those decision making processes. This has led some to argue that it is necessary to quantify patient preferences before they can be adequately considered.

A new framework for evaluating the health impacts of treatment for Gaucher disease type 1.

Background: The Disease Severity Scoring System (DS3) is a validated measure for evaluating Gaucher disease type 1 (GD1) severity. We developed a new framework, consisting of health states, transition probabilities between those states, and preferences for those states (utilities) based on the DS3 to predict long-term outcomes of patients starting treatment. We defined nine mutually exclusive (alive) health states based on three DS3 categories: mild (0 ≤ DS3 ≤ 3.