Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.

Background: Hypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade, an extended-release waxy maize cornstarch, was reported as an alternative to uncooked cornstarch (UCCS) which could prolong the duration of fasting in the GSD population. To date, there has been minimal published experience in (a) young children, (b) the ketotic forms of GSD, and (c) with daytime dosing.

'They don't know what to do with our children': Experiences and views on feeding and swallowing from parents of children who use long-term ventilation.

Increasing use of paediatric long-term ventilation (LTV) has been reported around the world over the last two decades and it is anticipated that use of this medical intervention will continue to grow. Research has shown that children who use LTV have risk factors for feeding and swallowing difficulties which result in long-term reliance on non-oral feeding methods. This Patient and Public Involvement (PPI) activity explored experiences of parents of children with LTV on their children's feeding and swallowing journeys.

A prospective cohort study exploring the impact of tonsillectomy on feeding difficulties in children.

Objectives: Paediatric feeding difficulties are common, affecting up to 25% of otherwise healthy children, symptoms include food refusal, gagging, choking, and excessive mealtime duration. These symptoms are commonly described in pre-operative discussions about tonsillectomy. This prospective study explores the impact of tonsillectomy on paediatric feeding difficulties.

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.

Background: Spinal muscular atrophy (SMA) is a progressive neuromuscular disease caused by mutations in Survival motor neuron 1 (SMN1) gene, leading to reduction in survival motor neuron protein (SMN), key for motor neuron survival and function in the brainstem and spinal cord. Risdiplam is an orally administered SMN2-splicing modifier which increases production of functional SMN protein. Risdiplam was offered in the UK under early access to medicines scheme (EAMS) to SMA type 1 and 2 patients aged 2 months and older, not suitable for authorised treatments from September 2020 to December 2021.

Prevalence of Pediatric Masked Hypertension and Risk of Subclinical Cardiovascular Outcomes: A Systematic Review and Meta-Analysis.

Masked hypertension (MH) occurs when office blood pressure is normal, but hypertension is confirmed using out-of-office blood pressure measures. Hypertension is a risk factor for subclinical cardiovascular outcomes, including left ventricular hypertrophy, increased left ventricular mass index, carotid intima media thickness, and pulse wave velocity. However, the risk factors for ambulatory blood pressure monitoring defined MH and its association with subclinical cardiovascular outcomes are unclear.

Paediatric TB care in the United Kingdom.

Care of patients with paediatric TB is delivered in a variety of settings by different clinicians in the United Kingdom. Paediatric practices vary in size. Guidelines on managing children with TB differ in recommendations.

Disparities between determinants of impaired vascular structure and function in young people with primary hypertension: a systematic review.

Objective: Early effects of primary hypertension on arterial structure and function in children and young people (CYP) and their determinants remain elusive. We aimed to review independent determinants of carotid intima-media thickness (cIMT), carotid wall cross-sectional area (WCSA) and carotid-femoral pulse wave velocity (cfPWV) in CYP with primary hypertension.

Methods: We performed a systematic review of studies reporting multivariable analysis of cfPWV, cIMT and WCSA in CYP (up to 25 years of age) with primary hypertension.

Diagnostic Accuracy of QuantiFERON-TB Gold Plus Assays in Children and Adolescents with Tuberculosis Disease.

In 2016, a new interferon-gamma release assay, QuantiFERON-TB Gold Plus, was introduced. We conducted a cross-sectional multicenter study, involving 158 children and adolescents with tuberculosis disease. The overall sensitivity of the assay was 82.

Urodynamic Outcomes in Children after Single and Multiple Injections for Overactive and Low Compliance Neurogenic Bladder Treated with Abobotulinum Toxin A.

Purpose: Intradetrusor botulinum toxin is an established part of the treatment pathway for pediatric patients with neurogenic bladder. We determined the urodynamic effect of single and multiple administrations of abobotulinum toxin A in pediatric patients with neurogenic bladder, and determined the urodynamic efficacy of abobotulinum toxin A in low compliance vs overactive bladders.

Materials And Methods: We conducted a single center retrospective review of all pediatric patients with neurogenic bladder treated with abobotulinum toxin A.

Prenatally diagnosed congenital lung malformations-A long-term outcome study.

Aim: To report the natural history of a cohort of children with prenatally diagnosed congenital lung malformations (CLM) which we set out to manage expectantly.

Methods: Retrospective review of children born between 1995 and 2013 with a CLM identified on prenatal ultrasound. Prenatal ultrasound data were analyzed along with patient medical records, radiology, and pathology.

Large Calibre Self-Expanding Stents for Pulmonary Stenosis After the Arterial Switch, a Low-Risk Solution to a Low-Flow Situation.

Branch pulmonary artery stenosis is one of the most common complications late after the arterial switch operation. The pathophysiology of stenosis in these circumstances is very different to that encountered in with normally related great vessels. The LeCompte manoeuvre leaves the pulmonary arteries straddled and stretched over the aortic root which contributing significantly to the degree of stenosis encountered.

Aquatic therapy for boys with Duchenne muscular dystrophy (DMD): an external pilot randomised controlled trial.

Background: Standard treatment of Duchenne muscular dystrophy (DMD) includes regular physiotherapy. There are no data to show whether adding aquatic therapy (AT) to land-based exercises helps maintain motor function. We assessed the feasibility of recruiting and collecting data from boys with DMD in a parallel-group pilot randomised trial (primary objective), also assessing how intervention and trial procedures work.

Device closure of secundum atrial septal defect's and the risk of cardiac erosion.

Cardiac erosion related to transcatheter atrial septal defect closure devices is of increasing concern. Erosion is reported to have occurred with most of currently available occluder devices. Perhaps due to the very large number of implants worldwide, the Amplatzer (St Jude) occluder is associated with the majority of cardiac erosion events reported in the literature.