648 research outputs found

    CFTR Modulator Therapy For Cystic Fibrosis

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    IntroductionCystic fibrosis (CF) is the most common life-limiting autosomal recessive condition in Caucasians, affecting the respiratory system, digestive tract and all exocrine glands. It is caused by a mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The genetic analysis, in addition to diagnosis, serves to create a modern individual CFTR modulator therapy.Aim: The aim of this article is to do a presentation of products for CFTR modulator therapy, which are available worldwide; introduction to their pharmacokinetic and pharmacodynamic properties; evaluation of the effect of their application.Materials: We have conducted a study of medical literature related to the products for CFTR modulator therapy and review of information on the topic.Results: The use of approved CFTR modulator therapy products in patients suitable for their use (specific genotype) results in an improvement in FEV1 values and body mass index and a reduction in the incidence of exacerbations in these patients.Conclusion: CFTR modulator therapy significantly improves the prognosis and quality of life of cystic fibrosis patients as a result of certain mutations in the CFTR gene. Quality care for other patients with other mutations is especially important so that they can benefit from personalized treatment in the future

    Exploring vertical and horizontal leadership in projects: A comparison of Indian and Australian contexts

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    Project-based organisational forms are becoming more and more prevalent in many industries, and leadership influences projects' success ultimately impacting the organisational performance. Two types of leadership styles have been explored: vertical and horizontal. This study aims to identify the nature and balance of vertical and horizontal leadership in projects to allow project managers to consciously poly these approaches in different situations. A case study-based approach is adopted wherein, two case studies from India and three case studies from Australia are included . A comparative study of leadership styles is performed to find the best contextual fit for leadership styles. The findings reveal that that national cultural is not a major factor in influencing project leadership. Rather, organisational culture and a shared understanding on leadership practices is what influences whether vertical or horizontal leadership will be more prevalent. Senior leaders' initiatives to create and support a culture of sharing ideasand decisions, backed by project manager's approach enable effective balance between horizontal and vertical leadership. Horizontal leadership is further by regular meetings and social interactions. Prevalence of horizontal leaderships is demonstrated in technical decisions, as team members have the best expertise to address technical issues. In contrast, strategic decisions are normally discussed with the project manager and often escalated to senior leaders for decisions

    Local risk proneness in analytically approximated utility functions under monotonically decreasing preferences

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    We discuss an analytical arctan form to approximate decreasing utilitiesbased on several nodes of its graphics elicited in interval form. We demonstratethe process on two types of nodes originating from different subjective elicitation approaches. Our focus is also on the local risk attitude estimator, whichin the case of decreasing preferences gets interpreted as local risk proneness vsthe local risk aversion for increasing preferences

    Health risk assessment of engine exhaust emissions within Australian ports: a case study of Port of Brisbane

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    Emissions from ocean-going vessels present a significant health risk to populations surrounding ports and damage the environment. Emissions from ships using heavy fuel oil include substantial amounts of sulphur dioxide, nitrogen oxides, and particulate matter. In order to assess the risk of these emissions, a complete methodology has been developed, based on the Australian Environmental Health Risk Assessment Framework. The method includes a detailed inventory of in-port and at-sea emissions using an activity-based approach applying downwash and near-field areas from first principles equations as well as the air-shed regions from CALPUFF dispersion modeling results for Port of Brisbane in 2013. The final risk values are validated against national and European guidelines. Various health impact assessments, as well as carcinogenic and ecological effects, are discussed in depth. This study offers a significant contribution to developing a baseline measurement of the current state of risk from emissions of the ocean-going vessels visiting the port, and suggests that, given the expected development of many Australian ports in the near future, the need for continual monitoring of shipping emissions is an essential and necessary area of research
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