14 results match your criteria: "Murdoch Children's Institute[Affiliation]"

Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.

Lancet Respir Med

December 2024

Population Policy and Practice Department, UCL Great Ormond Street Institute of Child Health, University College London, London, UK; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. Electronic address:

Article Synopsis
  • Vanzacaftor-tezacaftor-deutivacaftor is a new CFTR modulator showing safety and effectiveness in phase 2 trials for adults with cystic fibrosis, leading to a study evaluating its use in children aged 6-11.
  • This phase 3 trial, called RIDGELINE, involved participants from 33 clinical sites across eight countries, focusing on children with specific CFTR variants and stable health conditions.
  • The study aimed to assess the drug's safety, tolerability, and efficacy over 24 weeks, with primary outcomes evaluated through various health metrics and participant feedback.
View Article and Find Full Text PDF

Impact of Aortic Atresia After Fontan Operation in Patients With Hypoplastic Left Heart Syndrome.

Ann Thorac Surg

July 2023

Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Victoria, Australia; Department of Pediatrics, University of Melbourne, Melbourne, Victoria, Australia; Heart Research Group, Murdoch Children's Research Institute, Melbourne, Victoria, Australia; Melbourne Center for Cardiovascular Genomics and Regenerative Medicine, Murdoch Children's Institute, Melbourne, Victoria, Australia. Electronic address:

Background: Patients with aortic atresia have the worst prognosis in the spectrum of hypoplastic left heart syndrome. It remains unknown whether patients with aortic atresia continue to do poorly after Fontan operation. This study aimed to determine the association between aortic atresia and atrioventricular valve (AVV) function and clinical outcomes after Fontan operation in patients with hypoplastic left heart syndrome.

View Article and Find Full Text PDF

Importance: Exposure to early-life adversity alters the structural development of key brain regions underlying neurodevelopmental impairments. The association between prenatal exposure to adversity and brain structure at birth remains poorly understood.

Objective: To examine whether prenatal exposure to maternal social disadvantage and psychosocial stress is associated with neonatal global and regional brain volumes and cortical folding.

View Article and Find Full Text PDF

Ross Procedures in Children With Previous Aortic Valve Surgery.

J Am Coll Cardiol

September 2020

Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Victoria, Australia; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia; Heart Research Group, Murdoch Children's Research Institute, Melbourne, Victoria, Australia; The Melbourne Centre for Cardiovascular Genomics and Regenerative Medicine, Murdoch Children's Institute, Melbourne, Victoria, Australia. Electronic address:

Background: The Ross procedure in children is performed either as a primary operation, or a secondary operation after initial aortic valve surgery.

Objectives: The study aimed to determine whether the outcomes of primary and secondary Ross procedure are similar.

Methods: All patients who underwent Ross procedure between 1995 and 2018 were included in the study.

View Article and Find Full Text PDF

A comparison of videolaryngoscopy using standard blades or non-standard blades in children in the Paediatric Difficult Intubation Registry.

Br J Anaesth

January 2021

Department of Anesthesiology, Critical Care and Pain Medicine, Boston Children's Hospital, Boston, MA, USA; Department of Anaesthesia, Harvard Medical School, Boston, MA, USA.

Background: The design of a videolaryngoscope blade may affect its efficacy. We classified videolaryngoscope blades as standard and non-standard shapes to compare their efficacy performing tracheal intubation in children enrolled in the Paediatric Difficult Intubation Registry.

Methods: Cases entered in the Registry from March 2017 to January 2020 were analysed.

View Article and Find Full Text PDF

In the context of the current global COVID-19 pandemic, this Consensus Statement provides current recommendations for patients with, or at risk of developing, genetic heart disease, and for their health care management and service provision in Australia and New Zealand. Apart from general recommendations, there are specific recommendations for the following conditions: cardiomyopathy, Brugada syndrome (including in children), long QT syndrome (LQTS) and catecholaminergic polymorphic ventricular tachycardia (CPVT). Other recommendations are relevant to patient self-care and primary health care.

View Article and Find Full Text PDF

Utilizing three dimensional clinical gait analysis to optimize mobility outcomes in incomplete spinal cord damage.

Gait Posture

October 2019

Faculty of Medicine, Nursing and Allied Health Sciences, Monash University, VIC, 3800, Australia; Spinal Rehabilitation Service, Caulfield Hospital, Alfred Health, Caulfield, VIC, 3162, Australia; Rehabilitation and Aged Services Program, Department of Medicine, Monash Health, Cheltenham, VIC, 3192, Australia; Epworth-Monash Rehabilitation Medicine Unit, Monash University, VIC, 3800, Australia. Electronic address:

Background: Three-dimensional gait analysis (3DGA) has not previously been considered by consensus panels of spinal cord experts for use in studies of patients with spinal cord damage (SCD), yet it is frequently used in other neurological populations, such as stroke and cerebral palsy.

Research Question: How does 3DGA impairment based reporting guide individualised clinical decision-making in people with incomplete SCD?

Methods: Retrospective open cohort case series recruited 48 adults with incomplete SCD (traumatic or non-traumatic spinal cord dysfunction) referred to the Clinical Gait Analysis Service (CGAS), Melbourne, Australia. Three-dimensional gait data were used to identify gait impairments by the multidisciplinary clinical team.

View Article and Find Full Text PDF

A case-control genome-wide association study of ADHD discovers a novel association with the tenascin R (TNR) gene.

Transl Psychiatry

December 2018

School of Psychological Sciences and Monash Institute for Cognitive and Clinical Neurosciences (MICCN), Monash University, Melbourne, Australia.

It is well-established that there is a strong genetic contribution to the aetiology of attention deficit hyperactivity disorder (ADHD). Here, we employed a hypothesis-free genome-wide association study (GWAS) design in a sample of 480 clinical childhood ADHD cases and 1208 controls to search for novel genetic risk loci for ADHD. DNA was genotyped using Illumina's Human Infinium PsychArray-24v1.

View Article and Find Full Text PDF

Brain injury and altered brain growth in preterm infants: predictors and prognosis.

Pediatrics

August 2014

Department of Pediatric Newborn Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts; and

Background: To define the nature and frequency of brain injury and brain growth impairment in very preterm (VPT) infants by using MRI at term-equivalent age and to relate these findings to perinatal risk factors and 2-year neurodevelopmental outcomes.

Methods: MRI scans at term-equivalent age from 3 VPT cohorts (n = 325) were reviewed. The severity of brain injury, including periventricular leukomalacia and intraventricular and cerebellar hemorrhage, was graded.

View Article and Find Full Text PDF

Low risk of pulmonary valve implantation after a policy of transatrial repair of tetralogy of Fallot delayed beyond the neonatal period: the Melbourne experience over 25 years.

J Am Coll Cardiol

February 2014

Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Australia; Department of Pediatrics of the University of Melbourne, and the Murdoch Children's Institute, Melbourne, Australia.

Objectives: The study sought to evaluate the late outcomes of a policy of transatrial repair delayed beyond the neonatal period.

Background: Long-term outcomes of transatrial repair of tetralogy of Fallot are unknown.

Methods: The records of 675 consecutive patients undergoing a transatrial repair of tetralogy of Fallot between 1980 and 2005 were reviewed, their follow-up updated and survival confirmed from national death registries.

View Article and Find Full Text PDF

Aortic valve repair in children.

Ann Cardiothorac Surg

January 2013

Department of Cardiac Surgery, Royal Children's Hospital, Melbourne, Victoria, Australia; ; Department of Pediatrics of the University of Melbourne, and the Murdoch Children's Institute, Melbourne, Australia.

View Article and Find Full Text PDF

Background: The World Health Organization encourages the development of youth friendly services, yet little is known on how youth currently present in general practice.

Objective: To describe the perspectives, expectations, and service receipt of young people presenting to family doctors to inform the development of youth friendly services.

Design: Cross-sectional survey.

View Article and Find Full Text PDF

Performance of a whole blood interferon gamma assay for detecting latent infection with Mycobacterium tuberculosis in children.

Thorax

July 2006

Infectious Diseases Unit, Department of General Medicine and Murdoch Children's Institute, Royal Children's Hospital Melbourne, Flemington Road, Parkville 3052, Victoria, Australia.

Background: The diagnosis of latent Mycobacterium tuberculosis (MTB) infection with a tuberculin skin test (TST) in children is complicated by the potential influence of prior exposure to Bacille Calmette Geurin (BCG) vaccination or environmental mycobacteria. A whole blood assay has recently been developed to quantitatively measure interferon gamma (IFN-gamma) production by lymphocytes specific to the MTB antigens ESAT-6 and CFP-10, but its use and assessment in children has been limited. A study was undertaken to compare the performance of the whole blood IFN-gamma assay with the TST in diagnosing latent tuberculosis (TB) infection or TB disease in children in routine clinical practice.

View Article and Find Full Text PDF