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Systematic Literature Review of Access Pathways to Drugs for Patients with Rare Diseases.

Appl Health Econ Health Policy

December 2024

Centre for Health Economics Research and Evaluation, University of Technology Sydney, Level 5, Building 20, 100 Broadway, Chippendale, Sydney, NSW, 2008, Australia.

Objective: This article reviews the assessment pathways that have been implemented worldwide to facilitate access to drugs for patients with rare diseases.

Methods: The Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines were used to conduct a systematic literature review. The Ovid (Embase/MEDLINE), Cochrane, Web of Science, Econlit, National Institute of Health Research, Centre for Reviews and Dissemination, and International Network of Agencies for Health Technology Assessment databases were searched.

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Purpose: With the increasing demand for dialysis, there is a growing emphasis on patient-centered care. This study investigated patients' satisfaction levels with peritoneal dialysis (PD) and hemodialysis (HD) care in Iran.

Methods: A cross-sectional multicenter study was conducted among 346 patients with chronic kidney disease (CKD) covered by the Iran Health Insurance Organization who received dialysis services from October to December 2022 across the country.

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Objective(s): Some forms of breast cancer such as triple-negative phenotype, are serious challenge because of high metastatic cases, high mortality and resistance to conventional therapy motivated the search for alternative treatment approaches. Nanomaterials are promising candidates and suitable alternatives for improving tumor and cancer cell treatments.

Materials And Methods: Biosynthesis of ZnO NPs by help of Berberis integerrima fruit extract, has been done.

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Optimizing multireagent assays often requires successive titration of individual components until the optimal combination of conditions is achieved. This process is time-consuming, laborious, and often expensive since parallelized experimentation requires bulk consumption of reagents. Microfluidics presents a solution through miniaturization of standard processes by reducing reaction volume, executing multiple parallel workflows, and enabling automation.

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Realization of a sustainable hydrogen economy in the future requires the development of efficient and cost-effective catalysts for its production at scale. MXenes (MX) are a class of 2D materials with 'n' layers of carbon or nitrogen (X) interleaved by 'n+1' layers of transition metal (M) and have emerged as promising materials for various applications including catalysts for hydrogen evolution reaction (HER). Their properties are intimately related to both their composition and their atomic structure.

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