Background: Vosoritide is a C-type natriuretic peptide analog that addresses an underlying pathway causing reduced bone growth in achondroplasia. Understanding the vosoritide treatment effect requires evaluation over an extended duration and comparison with outcomes in untreated children.
Methods: After completing ≥6 months of a baseline observational growth study and 52 weeks in a double-blind, placebo-controlled study (ClinicalTrials.
Evidence-based assessment pathways inform early detection of cerebral palsy and access to intervention. This study investigated the relationships between early evidence-based assessments, diagnosis timeline, and rehabilitation intervention access in a population of children with cerebral palsy who were seen between 2010 and 2022 at the University of Wisconsin Waisman Center Newborn Follow Up Clinic. Cerebral palsy-specific assessments were increasingly integrated after the publication of early detection guidelines by Novak et al.
View Article and Find Full Text PDFIntroduction: Vosoritide is the first precision medical therapy approved to increase growth velocity in children with achondroplasia. Sharing early prescribing experiences across different regions could provide a framework for developing practical guidance for the real-world use of vosoritide.
Methods: Two meetings were held to gather insight and early experience from experts in Europe, the Middle East, and the USA.
Objective: The objective of this study was to describe the incidence and management of hydrocephalus in patients with achondroplasia over a 60-year period at four skeletal dysplasia centers.
Methods: The Achondroplasia Natural History Study (CLARITY) is a registry for clinical data from achondroplasia patients receiving treatment at four skeletal dysplasia centers in the US from 1957 to 2017. Data were entered and stored in a REDCap database and included surgeries with indications and complications, medical diagnoses, and radiographic information.
Int J Pediatr Otorhinolaryngol
December 2023
Cervical and craniocervical instability are associated with catastrophic procedural outcomes. We discuss three individuals who required otolaryngologic surgical intervention: two with symptomatic spinal instability and one in whom spinal stability was unable to be assessed. Two cases were managed with procedural positioning precautions and evoked potential monitoring, and the other with procedural positioning precautions alone.
View Article and Find Full Text PDFBackground: TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia.
Methods: ACcomplisH is a global, randomised, double-blind, placebo-controlled, dose-escalation trial.
Children with skeletal dysplasia are frequently referred to pediatric endocrinologists due to short stature. These children may present with disproportionate growth or medical histories that point to a skeletal dysplasia. This primer will discuss when to be concerned about skeletal dysplasia, the initial steps in evaluation for a skeletal dysplasia, and new therapies that are either recently approved or in development.
View Article and Find Full Text PDFBackground: The purpose of this study was to describe the frequency and risk factors for orthopedic surgery in patients with achondroplasia. CLARITY (The Achondroplasia Natural History Study) includes clinical data from achondroplasia patients receiving treatment at four skeletal dysplasia centers in the United States from 1957 to 2018. Data were entered and stored in a Research Electronic Data Capture (REDCap) database.
View Article and Find Full Text PDFPurpose: Pregnancies affected by maternal or fetal achondroplasia present unique challenges. The optimal route of delivery in fetuses with achondroplasia has not been established. Our objective was to determine whether the route of delivery affects postnatal achondroplasia-related surgical burden.
View Article and Find Full Text PDFGenomic studies often attempt to link natural genetic variation with important phenotypic variation. To succeed, robust and reliable phenotypic data, as well as curated genomic assemblies, are required. Wild sunflowers, originally from North America, are adapted to diverse and often extreme environments and have historically been a widely used model plant system for the study of population genomics, adaptation, and speciation.
View Article and Find Full Text PDFNational health strategies are integral in defining the vision and strategic direction for ensuring the health of a population or for a specific health area. To facilitate a national coordinated approach in spinal cord injury (SCI) research and care in Canada, Praxis Spinal Cord Institute, with support from national experts and funding from the Government of Canada, developed a national strategy to advance SCI care, health, and wellness based on previous SCI strategic documents. This paper describes the development process of the .
View Article and Find Full Text PDFBackground: Frail cardiac surgery patients have an increased risk of worse postoperative outcomes. The purpose of this study was to evaluate the implementation of a novel Telehealth Home monitoring Enhanced-Frailty And Cardiac Surgery (THE-FACS) intervention and determine its impact on clinical outcomes in frail patients post-cardiac surgery.
Methods: Frail/vulnerable patients defined by Edmonton Frailty Scale (EFS > 4) undergoing cardiac surgery were prospectively enrolled (November 2019 -March 2020) at the New Brunswick Heart Centre.
Background: Physical therapists (PTs) should know how to best treat patients with inflammatory arthritis.
Objective: To document interventions chosen by PTs for patients with rheumatoid arthritis (RA) and ankylosing spondylitis (AS) and whether choices follow evidence-based practice.
Methods: Licensed musculoskeletal PTs in Quebec, Canada responded to an online survey.
The province of Quebec, Canada is the most important organic cranberry growing region worldwide. However, insect pest management methods are limited, and growers face significant yield loss each year, mostly caused by lepidopteran pests. Egg parasitoids of the genus Trichogramma (Hymenoptera: Trichogrammatidae) are effective biocontrol agents of lepidopteran pests in many forests and agricultural systems.
View Article and Find Full Text PDFObjectives: This study aims to determine whether patients with active rheumatoid arthritis (RA), either starting on or changing biological or targeted synthetic disease-modifying antirheumatic drugs (DMARDs), demonstrate better self-management safety skills three months after receiving a multidisciplinary educational intervention compared to patients receiving usual care.
Patients And Methods: Between October 2015 and October 2018 , this open-label, randomized-controlled trial included a total of 107 RA patients (27 males, 80 females; mean age: 60.2±10.
Background: Globally, an increasing number of vulnerable or frail patients are undergoing cardiac surgery. However, large-scale frailty data are often limited by the need for time-consuming frailty assessments. This study aimed to (1) create a retrospective registry-based frailty score (FS), (2) determine its effect on outcomes and age, and (3) health care costs.
View Article and Find Full Text PDFThe coronavirus disease 2019 (COVID-19) pandemic continues to disrupt the provision of cardiac procedural services due to overwhelming interval surges in COVID-19 cases and the associated crisis of cardiac intervention deferment. Despite the availability of widespread testing, highly efficacious vaccines, and intensive public health efforts, the pandemic is entering its third year, where new severe acute respiratory syndrome-coronavirus-2 variants have increased the likelihood that patients scheduled for a cardiac intervention will contract COVID-19 in the perioperative period. The Society of Thoracic Surgeons (STS) Workforce on Critical Care, the STS Workforce on Adult Cardiac and Vascular Surgery, and the Canadian Society of Cardiac Surgeons have developed this document, endorsed by the STS and affirmed by the Society of Cardiovascular Angiography and Interventions and the Canadian Association of Interventional Cardiology, to provide guidance for cardiac procedure deferment and intervention timing for preoperative patients diagnosed with COVID-19.
View Article and Find Full Text PDFEarly referral to rheumatology of people with suspected inflammatory arthritis is associated with better outcomes. Typically, these individuals are seen by a family physician who would assess the need for rheumatology referral. However, some may first consult a physical therapist where no physician referral is required.
View Article and Find Full Text PDFBackground: While fast track clinical pathways have been demonstrated to reduce resource utilization in patients undergoing cardiac surgery, it remains unclear as to whether they adversely affect post-operative outcomes. The purpose of this study was to determine the impact of fast tracking on post-operative outcomes following cardiac surgery.
Methods: In a retrospective study, all patients undergoing first-time, on-pump, non-emergent coronary artery bypass grafting, valve, or coronary artery bypass grafting + valve at a single centre between 2010 and 2017 were included.
Canada is a wealthy nation with a geographically diverse population, seeking health innovations to better serve patients in accordance with the Canada Health Act. In this country, population and geography converge with social determinants, policy, procurement regulations, and technological advances with the goal to achieve equity in the management and distribution of health care. Rural and remote patients are a vulnerable population; when managing chronic conditions like cardiovascular disease, there is currently inequity to accessing specialist physicians at the recommended frequency-increasing the likelihood of poor health outcomes.
View Article and Find Full Text PDFVariation in floral displays, both between and within species, has been long known to be shaped by the mutualistic interactions that plants establish with their pollinators. However, increasing evidence suggests that abiotic selection pressures influence floral diversity as well. Here, we analyse the genetic and environmental factors that underlie patterns of floral pigmentation in wild sunflowers.
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