Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update.

Duchenne muscular dystrophy (DMD) afflicts 1 in 5000 newborn males, leading to progressive muscle weakening and the loss of ambulation between the ages of 8 and 12. Typically, DMD patients pass away from heart failure or respiratory failure. Currently, there is no cure, though exon-skipping therapy including eteplirsen (brand name Exondys 51), a synthetic antisense oligonucleotide designed to skip exon 51 of the dystrophin gene, is considered especially promising.

How can we better identify patients with rectal bleeding who are at high risk of colorectal cancer? An observational study.

Purpose: Rectal bleeding is a common symptom of colorectal cancer. In this paper, we describe and evaluate the operation of a central access and triage system for patients with rectal bleeding, which uses a "high-risk"/ "low-risk" designation based on the referring doctor's subjective designation and a 10-item symptom checklist.

Methods: A total of 1846 patients, referred between February 1, 2016, and December 31, 2018, were included.

Esthetic outcome for implant therapy of a maxillary lateral incisor using prefabricated titanium and customized zirconia abutments: 4-year clinical reports.

The reported clinical scenarios presented two patients, one managed with a prefabricated abutment and the other with a customized abutment, and both patients were pleased with the outcome. However, from a professional viewpoint, the esthetic outcome using the custom zirconia abutment was superior to that using the prefabricated titanium abutment.

What work is required to implement and sustain the National Surgical Quality Improvement Program (NSQIP)? A qualitative study of NSQIP implementation in Alberta, Canada.

Objectives: Hospitals introducing the National Surgical Quality Improvement Program (NSQIP) face implementation challenges. To understand the work of embedding NSQIP into routine practice, we explored interactions between contextual factors and the work among implementation teams at the individual, team and organisational level to illuminate how to support and sustain NSQIP implementation.

Design: Qualitative interpretative study using thematic analysis.

Impact of restricted visitation policies in hospitals on patients, family members and healthcare providers during the COVID-19 pandemic: a scoping review protocol.

Introduction: Flexible visitation policies in hospitals are an important component of care that contributes to reduced stress and increased satisfaction among patients and their family members. Early evidence suggests restricted visitation policies enacted in hospitals during the COVID-19 pandemic are having unintended consequences on patients, family members and healthcare providers. There is a need for a comprehensive summary of the impacts of restricted visitation policies on key stakeholders and approaches to mitigate that impact.

Current status of health systems financing and oversight for end-stage kidney disease care: a cross-sectional global survey.

Objectives: The Global Kidney Health Atlas (GKHA) is a multinational, cross-sectional survey designed to assess the current capacity for kidney care across all world regions. The 2017 GKHA involved 125 countries and identified significant gaps in oversight, funding and infrastructure to support care for patients with kidney disease, especially in lower-middle-income countries. Here, we report results from the survey for the second iteration of the GKHA conducted in 2018, which included specific questions about health financing and oversight of end-stage kidney disease (ESKD) care worldwide.

eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.

Exon skipping using antisense oligonucleotides (ASOs) has recently proven to be a powerful tool for mRNA splicing modulation. Several exon-skipping ASOs have been approved to treat genetic diseases worldwide. However, a significant challenge is the difficulty in selecting an optimal sequence for exon skipping.

Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy.

The debilitating neuromuscular disorders Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), which harm 1 in 5000 newborn males and 1 in 11,000 newborns, respectively, are marked by progressive muscle wasting among other complications. While DMD causes generalized muscle weakness due to the absence of the dystrophin protein, SMA patients generally face motor neuron degeneration because of the lack of the survival motor neuron (SMN) protein. Many of the most promising therapies for both conditions restore the absent proteins dystrophin and SMN.

A Strategic Program for Risk Assessment and Intervention to Mitigate Environmental Stressor-Related Adverse Pregnancy Outcomes in the Indian Population.

Global environmental stressors of human health include, but are not limited to, conflict, migration, war, natural disasters, climate change, pollution, trauma, and pandemics. In combination with other factors, these stressors influence physical and mental as well as reproductive health. Maternal stress is a known factor for adverse pregnancy outcomes such as preterm birth (PTB); however, environmental stressors are less well-understood in this context and the problem is relatively under-researched.

Impact of home telemonitoring and management support on blood pressure control in non-dialysis CKD: a systematic review protocol.

Introduction: Hypertension is a common public health problem and a key modifiable risk factor for cardiovascular (CV) and chronic kidney disease (CKD). Home blood pressure (BP) telemonitoring (HBPT) and management is associated with improved BP control, accelerated delivery of care and decision-making strategies that can reduce adverse outcomes associated with hypertension. The aim of this paper is to describe the protocol for a systematic review to assess the impact of HBPT interventions used for improving BP control and reducing CV and kidney outcomes in non-dialysis CKD patients.

Risk factors for severity of COVID-19: a rapid review to inform vaccine prioritisation in Canada.

Objectives: Rapid review to determine the magnitude of association between potential risk factors and severity of COVID-19, to inform vaccine prioritisation in Canada.

Setting: Ovid MEDLINE(R) ALL, Epistemonikos COVID-19 in L·OVE Platform, McMaster COVID-19 Evidence Alerts and websites were searched to 15 June 2020. Eligible studies were conducted in high-income countries and used multivariate analyses.

Sex-related differences in opioid administration in the emergency department: a population-based study.

Background: Sex differences in pain experience and expression may influence ED pain management. Our objective was to evaluate the effect of sex on ED opioid administration.

Methods: We conducted a multicentre population-based observational cohort study using administrative data from Calgary's four EDs between 2017 and 2018.

Salvage of Malfunctioning Peritoneal Dialysis Catheters: An Algorithm for Recanalization and Repositioning.

A peritoneal dialysis catheter salvage algorithm was developed and performed for 40 patients with documented catheter malfunction (obstruction and/or malposition) referred to the interventional radiology suite. This procedure utilized a metallic stiffener for repositioning and rotating dual guide wires for recanalization. A retrospective analysis of 35 cases of fluoroscopic manipulation showed that in 83% of the cases, the catheters were successfully repositioned and/or recanalized, and in 59%, they remained patent at 30 days.

Bilateral leg swelling as the presenting symptom of Löfgren syndrome in a paediatric patient: a rare presentation of a rare paediatric disease.

A 17-year-old previously healthy man presented with a 4-week history of progressive bilateral leg swelling with discomfort and erythema, but no signs of arthritis or erythema nodosum. An incidental finding of a query pulmonary nodule on chest X-ray prompted chest CT for further evaluation, revealing bilateral hilar and mediastinal lymphadenopathy. The patient then underwent endobronchial ultrasound and transbronchial needle aspiration biopsies of mediastinal lymph nodes.

Applying improvement science to establish a resident sustained quality improvement (QI) educational model.

Background: Prior to 2017, internal medicine (IM) residents at the University of Alberta did not have a standardised quality improvement (QI) educational curriculum. Our goal was to use QI principles to develop a resident sustained curriculum using the Evidence-based Practice for Improving Quality (EPIQ) training course.

Methods: Three one-year Plan-Do-Study-Act (PDSA) cycles were conducted.

Identification and operationalisation of indicators to monitor successful uptake of advance care planning policies: a modified Delphi study.

Background: In 2014, the province of Alberta, Canada implemented a province-wide policy and procedures for advance care planning (ACP) and goals of care designation (GCD) across its complex, integrated public healthcare system. This study was conducted to identify and operationalise performance indicators for ACP/GCD to monitor policy implementation success and sustainment of ACP/GCD practice change.

Methods: A systematic review and environmental scan was conducted to identify potential indicators of ACP/GCD uptake (n=132).

Workforce capacity for the care of patients with kidney failure across world countries and regions.

Introduction: An effective workforce is essential for optimal care of all forms of chronic diseases. The objective of this study was to assess workforce capacity for kidney failure (KF) care across world countries and regions.

Methods: Data were collected from published online sources and a survey was administered online to key stakeholders.

Community engagement approaches for Indigenous health research: recommendations based on an integrative review.

Objective: Community engagement practices in Indigenous health research are promoted as a means of decolonising research, but there is no comprehensive synthesis of approaches in the literature. Our aim was to assemble and qualitatively synthesise a comprehensive list of actionable recommendations to enhance community engagement practices with Indigenous peoples in Canada, the USA, Australia and New Zealand.

Design: Integrative review of the literature in medical (Medline, Cumulative Index to Nursing and Allied Health Literature and Embase) and Google and WHO databases (search cut-off date 21 July 2020).

Points to consider for the treatment of immune-mediated inflammatory diseases with Janus kinase inhibitors: a consensus statement.

Objectives: Janus kinase inhibitors (JAKi) have been approved for use in various immune-mediated inflammatory diseases. With five agents licensed, it was timely to summarise the current understanding of JAKi use based on a systematic literature review (SLR) on efficacy and safety.

Methods: Existing data were evaluated by a steering committee and subsequently reviewed by a 29 person expert committee leading to the formulation of a consensus statement that may assist the clinicians, patients and other stakeholders once the decision is made to commence a JAKi.

Delphi developed syllabus for the medical specialty of sport and exercise medicine: part 2.

Training in the medical specialty of sport and exercise medicine (SEM) is available in many, but not all countries. In 2015, an independent Delphi group, the International Syllabus in Sport and Exercise Medicine Group (ISSEMG), was formed to create a basic syllabus for this medical specialty. The group provided the first part of this syllabus, by identifying 11 domains and a total of 80 general learning areas for the specialty, in December 2017.

Perioperative management for people with chronic kidney disease receiving dialysis undergoing major surgery: a protocol for a scoping review.

Introduction: People with chronic kidney disease receiving dialysis (CKD G5D) have an increased risk of poor postoperative outcomes and a high incidence of major surgery. Despite the high burden of these combined risks, there is a paucity of evidence to support tailored perioperative strategies to manage this population. A comprehensive evidence synthesis would inform the management of these patients in the perioperative period and identify knowledge gaps.