A5041811009- Congenital Diaphragmatic Hernia Studies
- Muscle Physiology and Disorders
- Neurogenetic and Muscular Disorders Research
- Scoliosis diagnosis and treatment
- Respiratory Support and Mechanisms
- Chronic Obstructive Pulmonary Disease (COPD) Research
- Neonatal Respiratory Health Research
- Atomic and Subatomic Physics Research
- Pleural and Pulmonary Diseases
- Pectus Deformity Diagnosis and Treatment
- Spinal Fractures and Fixation Techniques
- Cystic Fibrosis Research Advances
- Congenital Anomalies and Fetal Surgery
- Advanced Sensor and Energy Harvesting Materials
- Trauma Management and Diagnosis
- Mechanical Circulatory Support Devices
- Cardiomyopathy and Myosin Studies
- Children's Physical and Motor Development
- Palliative Care and End-of-Life Issues
- Neuroscience of respiration and sleep
- Childhood Cancer Survivors' Quality of Life
- Tracheal and airway disorders
- Cardiac electrophysiology and arrhythmias
- Hip disorders and treatments
- Advanced MRI Techniques and Applications
Children's Hospital of Philadelphia
2016-2025
University of Pennsylvania
2009-2024
Oregon Health & Science University
2024
Boston Children's Hospital
2024
Evelina London Children's Healthcare
2024
University College London
2020-2024
Great Ormond Street Hospital
2020-2024
Royal Brompton Hospital
2024
Society for Classical Studies
2021
University of Florida
2019
Decline in pulmonary function Duchenne Muscular Dystrophy (DMD) contributes to significant morbidity and reduced longevity. Spirometry is a widely used fairly easily performed technique assess lung function, particular volume; however, the acceptability criteria from American Thoracic Society (ATS) may be overly restrictive inappropriate for patients with neuromuscular disease. We examined prospective spirometry data (Forced Vital Capacity [FVC] peak expiratory flow [PEF]) 60 DMD enrolled...
BackgroundCardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety efficacy sequential intravenous infusions human allogeneic CDCs late-stage dystrophy.MethodsIn this multicentre, randomised, double-blind, placebo-controlled, phase 2 trial, patients with dystrophy, aged 10 years or older moderate upper limb impairment, were enrolled at seven centres USA. Patients randomly...
To describe the respiratory trajectories and their correlation with motor function in an international pediatric cohort of patients type 2 nonambulant 3 spinal muscular atrophy (SMA).This was 8-year retrospective observational study International SMA Consortium (iSMAc) natural history study. We retrieved anthropometrics, forced vital capacity (FVC) absolute, FVC percent predicted (FVC%P), noninvasive ventilation (NIV) requirement. Hammersmith Functional Motor Scale (HFMS) revised Performance...
Background: The vertical expandable prosthetic titanium rib (VEPTR) has been inserted in children with thoracic insufficiency syndrome for the last decade to expand and support chest allow further lung growth. There are minimal published data evaluating postoperative change function after VEPTR insertion. We hypothesize that there will be a significant increase insertion, earlier greater improvement. Methods: Chest Wall Disorders Study Group Database, containing before insertion from 7...
Spine and chest wall deformities in children with early onset scoliosis (EOS) frequently impair respiratory function postnatal growth of the lung. While a relationship between deformity such impairment has been reported adolescent idiopathic it is not well understood early-onset (EOS).We therefore describe (1) preoperative relation Cobb angle forced vital capacity (FVC) infants EOS; (2) how changes before after surgery relate to lung ventilation perfusion right left lungs.We measured FVC 10...
Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle loss and premature death, most commonly from respiratory or cardiac failure. DMD primarily caused by whole exon deletions, resulting shift of the dystrophin mRNA reading frame prevents production functional protein. Eteplirsen, phosphorodiamidate morpholino oligomer (PMO), designed to skip 51, restore frame, induce internally shortened patients with mutations amenable such...
Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study 10–18 year old DMD the assessment inter-correlation between tests (PFTs), their reliability association general disease stage measured by Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], volume one second [FEV1]) maximum static airway pressures (MIP, MEP) were prospectively collected from 64 enrolled...
Summary Assessment of dynamic inspiratory function may provide valuable information about the degree and progression pulmonary involvement in patients with Duchenne muscular dystrophy (DMD). The aims this study were to characterize assess efficacy idebenone on outcome a large well‐characterized cohort 10–18 year‐old DMD not taking glucocorticoid steroids (GCs) enrolled phase 3 randomized controlled DELOS trial. We evaluated effect highest flow generated during an FVC maneuver (maximum flow;...
Background Available methods to quantify regional dynamic thoracic function in insufficiency syndrome (TIS) are limited. Purpose To evaluate the use of quantitative MRI depict changes before and after surgical correction TIS. Materials Methods Images from free-breathing pediatric patients with TIS (July 2009-August 2015) were retrospectively evaluated by using vertical expandable prosthetic titanium rib (VEPTR). Eleven volumetric parameters derived lung, chest wall, diaphragm segmentations,...
Decline in respiratory function patients with DMD starts during early teenage years and leads to morbidity mortality. Published evidence of efficacy for idebenone on outcomes is currently limited 12 months follow-up time. Here we report data collected as retrospective cohort study (SYROS) from 18 not using glucocorticoids who were treated (900 mg/day) under Expanded Access Programs (EAPs). The objective was assess the long-term evolution periods On-Idebenone compared Off-Idebenone same...
Nusinersen has shown significant functional motor benefit in the milder types of spinal muscular atrophy (SMA). Less is known on respiratory outcomes patients with nusinersen-treated SMA. The aim this study was to describe changes function pediatric SMA type 2 and 3 regular treatment nusinersen within iSMAc international cohort compare their trajectory natural history (NH) data published by consortium 2020.
Purpose To investigate free-breathing thoracic bright-blood four-dimensional (4D) dynamic MRI (dMRI) to characterize aeration of parenchymal lung tissue in healthy children and patients with insufficiency syndrome (TIS). Materials Methods All dMR images TIS were collected from July 2009 June 2017. Standardized signal intensity (sSI) was investigated, first using a phantom establish feasibility sensitivity then retrospective research study 40 (16 male, 24 female; mean age, 9.6 years ± 2.1...
We provide evidence for an immediate effect of NG-monomethyl-L-arginine (L-NMMA) on the force-frequency relationship in isolated hamster papillary muscles. L-NMMA (competitive inhibitor nitric oxide synthase) reversed (staircase effect) muscles from negative to positive (P < .01; ANOVA; n = 6). The addition L-arginine (substrate blocked (NO) donor, sodium nitroprusside (NTP), significantly increased level cGMP tissue bath test; 6) and inotropic staircase 8-Br-cGMP resulted a...
Abstract Rationale Conducting clinical trials in cystic fibrosis (CF) preschoolers has been limited by lack of sensitive lung function measures performed across sites. Objectives (1) Assess feasibility and short‐term reproducibility spirometry, forced oscillometry (FO), inductance plethysmography (IP) a multi‐center preschool population; (2) compare ability each technique to differentiate CF controls; (3) evaluate longitudinal changes function; (4) estimate sample sizes for future trials....