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

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.

Trends in the surgical management of proximal humerus fractures over the last 20 years from Australian registry databases.

Background: Proximal humerus fractures (PHF) are common with approximately 30% requiring surgical intervention. This ranges from open reduction internal fixation (ORIF) to shoulder arthroplasty (including hemiarthroplasty, total shoulder arthroplasty (TSA), reverse total shoulder arthroplasty (RTSA)). The aim of this study was to assess trends in operative interventions for PHF in an Australian population.

Effect of Qigong exercise on quality of life and cortisol in patients with cancer: a non-randomized controlled trial.

Purpose: The aim of this study was to investigate the effect of a 6-month Guolin Qigong on quality of life and serum cortisol among patients with cancer.

Methods: This study was a two-arm and non-randomized controlled trial. Forty-nine patients with cancer who were over 18 years of age and diagnosed with cancer were enrolled in this study and assigned to either the Guolin Qigong intervention group (IG, n = 26) or the usual care group (UC, n = 23) for 6 months.

Latent Tuberculosis Infection Amongst Allogeneic Hematopoietic Stem Cell Transplant Recipients: The Impact of Routine Pretransplant Review by a Transplant Infectious Diseases Physician.

Background: Identifying patients with latent tuberculosis infection (LTBI) is challenging. This is particularly true amongst immunocompromised hosts, in whom the diagnostic accuracy of available tests is limited. The authors evaluated the impact of routine pretransplant review by a transplant infectious diseases (TID) physician on LTBI screening in allogeneic hematopoietic stem cell transplant (alloHSCT) recipients.

Historical, Theoretical and Methodological Foundations of CHAT: Critical Deliberations.

The paper critically analyses cultural-historical activity theory (CHAT) in two most important aspects - the basic unit of analysis (i.e., in essence, the subject of the theory) and the main epistemological and methodological principles.

Robot-Assisted Laparoscopic Sacrohysteropexy with Autologous Fascia Lata.

Introduction And Hypothesis: Autologous fascia lata has been increasingly utilised in pelvic floor reconstructive surgeries such as sacrocolpopexy and sacrohysteropexy. This case highlights sacrohysteropexy with autologous fascia lata as a promising option for women with advanced uterovaginal prolapse who wish to preserve their uterus and avoid synthetic mesh.

Methods: We report the case of a 65-year-old woman with stage 3 pelvic organ prolapse following one forceps and one spontaneous vaginal delivery.

A systematic review on ethnobotany, phytochemistry, and pharmacology of R. Br. (Boraginaceae), a medicinal plant.

For the first time, critical review on R. Br. (Boraginaceae) is established.

Improved Control of Triple-Negative Breast Cancer Tumor and Metastasis with a pH-Sensitive Hyaluronic Acid Nanocarrier for Doxorubicin Delivery.

Polymer based nanoformulations offer substantial prospects for efficacious chemotherapy delivery. Here, we developed a pH-responsive polymeric nanoparticle based on acidosis-triggered breakdown of boronic ester linkers. A biocompatible hyaluronic acid (HA) matrix served as a substrate for carrying a doxorubicin (DOX) prodrug which also possesses natural affinity for CD44 cells.

High-Performance Shear Mode Ultrasonic Transducer Operating at Ultrahigh Temperature Fabricated with BiSiO Piezoelectric Crystal.

Shear mode ultrasonic waves are in high demand for structural health monitoring (SHM) applications owing to their nondispersive characteristics, singular mode, and minimal energy loss, especially in harsh environments. However, the generation and detection of a pure shear wave using conventional piezoelectric materials present substantial challenges because of their complex piezoelectric response, involving multiple modes. Herein, we introduce a high-quality piezoelectric crystal BiSiO (BSO), exhibiting a robust piezoelectric response ( = 45.

Outliers of Treatment Frequency in Retinal Vein Occlusion: 24-Month Comparative Analysis of Fight Retinal Blindness! Practitioners.

Background: We aimed to describe a 2-year outcome of eyes managed by practitioners benchmarked using a funnel plot by their frequency of treatment using vascular endothelial growth factor (VEGF) inhibitors for naive retinal vein occlusion (RVO).

Methods: A multicentre, international, observational study of 29 doctors in 12 countries managing 1110 eyes with RVO commencing VEGF inhibitors between 1 January 2012-2022 tracked in the Fight Retinal Blindness! registry.

Results: We identified 3 outlying 'intensive' practitioners (managing 350/1110 eyes [32%]), 22 'typical' practitioners (604/1110, [54%]) and 4 outlying 'relaxed' practitioners (156/1110, [14%]) with respective 24-month outcomes in Branch and Central RVO including the primary outcome, mean adjusted change in visual acuity (VA) in BRVO: +16.

Impact of Race on the Outcomes of Retinoblastoma Treated With Primary Enucleation: A Global Study of 1426 Patients.

Background: To evaluate the clinical presentation, pathological features and outcomes of retinoblastoma based on the race of origin in a global cohort of patients.

Methods: Retrospective collaborative study of 1426 patients who underwent primary enucleation for retinoblastoma.

Results: Patients were grouped into Caucasians (n = 231, 16%), Asians (n = 841, 59%), Hispanics (n = 226, 16%), Arabs (n = 96, 7%) and Others (Africans, African Americans, Indigenous Australians; n = 32, 2%) cohorts.

Development of a Self-Deploying Extra-Aortic Compression Device for Medium-Term Hemodynamic Stabilization: A Feasibility Study.

Hemodynamic stabilization is crucial in managing acute cardiac events, where compromised blood flow can lead to severe complications and increased mortality. Conditions like decompensated heart failure (HF) and cardiogenic shock require rapid and effective hemodynamic support. Current mechanical assistive devices, such as intra-aortic balloon pumps (IABP) and extracorporeal membrane oxygenation (ECMO), offer temporary stabilization but are limited to short-term use due to risks associated with prolonged blood contact.

A comparison of the accuracy and feasibility of a low-cost mobile application versus higher-cost handheld 3D scanner for digital ear prosthetics.

Background: Facial prosthetics are an important means to rehabilitate patients with congenital or acquired facial defects. However, with a time-consuming manual workflow and workforce shortage, access to facial prosthetics is limited in Australia and worldwide, especially for rural and remote patients. Optical 3D scanning has been increasingly integrated in digitizing data.

Novel Insights into Enhanced Stability of Li-Rich Layered and High-Voltage Olivine Phosphate Cathodes for Advanced Batteries through Surface Modification and Electron Structure Design.

The design of cathode/electrolyte interfaces in high-energy density Li-ion batteries is critical to protect the surface against undesirable oxygen release from the cathodes when batteries are charged to high voltage. However, the involvement of the engineered interface in the cationic and anionic redox reactions associated with (de-)lithiation is often ignored, mostly due to the difficulty to separate these processes from chemical/catalytic reactions at the cathode/electrolyte interface. Here, a new electron energy band diagrams concept is developed that includes the examination of the electrochemical- and ionization- potentials evolution upon batteries cycling.

Potential Evolutionary Convergence in Trophic Adaptations of Two Booidean Snake Lineages as Evidenced by Skull Morphology.

Booidean snakes are a diverse and widespread lineage with an intriguing evolutionary and biogeographic history. By means of cranial morphology and osteology, this study investigates the evolutionary convergence in the Neotropical genera Boa and Corallus on the one hand and the Malagasy clade comprising Acrantophis and Sanzinia on the other. We hypothesize that the mostly arboreal Corallus and Sanzinia present larger jaws and longer teeth to keep hold of the prey and resist gravity and torsional forces acting on their skull while hanging from branches, while terrestrial genera such as Acrantophis show thinner jaws with shorter teeth because they can rely on the full length of their coils to immobilize and constrict the prey together with a substrate that supports the whole of their body.

Pathways From Spatial Skills to Mathematics: The Roles of Gender and Fluid Reasoning.

There are contentious and persistent gender differences reported in some measures of spatial skills, particularly mental rotation and, to a lesser extent, perspective-taking, which may have an impact on mathematics success. Furthermore, pathways between spatial skills and mathematics may be mediated by other cognitive factors, such as fluid reasoning. Participants (N = 320, age range 8-12 years) completed measures of mental rotation, perspective-taking, fluid reasoning, and mathematics.

Perspectives of current and future use of electrical stimulation home-devices from people with spinal cord injuries and healthcare professionals.

Introduction: Electrical stimulation (E-stim) can reduce the impact of complications, like spasticity, bladder dysfunction in people with spinal cord injuries (SCIs), enhancing quality of life and health outcomes. With SCI prevalence high in regional Australia and a shift towards home-based community integrated care, the perspectives of people with SCI and healthcare professionals on current and future use of E-stim home-devices are needed.

Methods: A mixed-methods concurrent triangulation approach was used.

Exploring therapeutic alliance in spinal cord injury rehabilitation: Control, identity, and liminality.

Background: The therapeutic alliance (TA) has emerged as a key principle in enhancing the quality of health and rehabilitation services.

Purpose: This study aimed to explore patient perspectives on the influence of TAs with practitioners during inpatient rehabilitation following spinal cord injury (SCI).

Methods: Using a qualitative methodology, ( = 18) in-depth interviews were conducted with inpatient participants.

Tranexamic acid in hip and spine surgery for children with cerebral palsy - a PRISMA-compliant scoping review.

Many children with cerebral palsy (CP) are frail and require major hip and/or spine surgeries associated with substantial blood loss. Tranexamic acid (TXA) is commonly used to reduce blood loss, but there is uncertainty around the optimal dose and timing of administration. There have been reviews in sub-populations and specific dosing regimens, but a broad overview of the available literature is lacking.

Forecast of total health expenditure on China's ageing population: a system dynamics model.

Background: China is currently at a turning point as its total population has started to decline, and therefore faces issues related to caring for an ageing population, which will require an increase in Total Health Expenditure (THE). Therefore, the ability to forecast China's future THE is essential.

Methods: We developed two THE System Dynamics (SD) models using Stella Architect 3.

Agrobacterium-mediated transient transformation of Flaveria bidentis leaves: a novel method to examine the evolution of C photosynthesis.

The genus Flaveria has been studied extensively as a model for the evolution of C photosynthesis. Thus far, molecular analyses in this genus have been limited due to a dearth of genomic information and the lack of a rapid and efficient transformation protocol. Since their development, Agrobacterium-mediated transient transformation protocols have been instrumental in understanding many biological processes in a range of plant species.

The Scottish Simulation 'KSDP' Design Framework: a sense-making and ordered approach for building aligned simulation programmes.

Impactful learning through simulation-based education involves effective planning and design. This can be a complex process requiring educators to master a varied toolkit of analysis tools, learning methodologies, and evaluative strategies; all to ensure engagement of learners in a meaningful and impactful way. Where there is a lack of thoughtful design, simulation-based education programmes may be inefficiently deployed at best, and completely ineffective or even harmful to learning and learners at worst.

Rare bilateral vascular variations of the upper limb: a cadaveric case study.

Arterial variations in the upper limb are of significant clinical importance, especially in procedures such as venepunctures, coronary artery bypass grafts, trauma reconstructive surgeries, brachial plexus nerve blocks, and breast reconstructions. This report presents previously undocumented arterial variations in the upper limbs in a 95-year-old female cadaveric donor. We observed bilateral superficial ulnar arteries originating at the cubital fossa, deviating from the previously reported origin at the proximal brachial artery.