- Health Systems, Economic Evaluations, Quality of Life
- Parathyroid Disorders and Treatments
- Magnesium in Health and Disease
- Gastrointestinal Bleeding Diagnosis and Treatment
- Dialysis and Renal Disease Management
- Central Venous Catheters and Hemodialysis
- Cardiovascular Function and Risk Factors
- Adolescent and Pediatric Healthcare
- Pluripotent Stem Cells Research
- Immunodeficiency and Autoimmune Disorders
- Intestinal Malrotation and Obstruction Disorders
- Pregnancy and Medication Impact
- Cell Adhesion Molecules Research
- Restless Legs Syndrome Research
- Genetic Syndromes and Imprinting
- Digestive system and related health
- Immune Response and Inflammation
- Renal and related cancers
- Neutrophil, Myeloperoxidase and Oxidative Mechanisms
- Heart Failure Treatment and Management
- Bone health and osteoporosis research
- Climate Change and Health Impacts
- Statistical Methods in Clinical Trials
- Eosinophilic Esophagitis
- Pediatric Hepatobiliary Diseases and Treatments
Children's Hospital of Philadelphia
2023
University of Massachusetts Chan Medical School
2023
Lawson Health Research Institute
2021
Boston Children's Hospital
2019-2020
London Health Sciences Centre
2020
Harvard University
2019-2020
Background & AimsMutations in the tetratricopeptide repeat domain 7A gene (TTC7A) cause intestinal epithelial and immune defects. Patients can become deficient develop apoptotic enterocolitis, multiple atresia, recurrent stenosis. The disease patients with TTC7A deficiency is severe untreatable, it recurs despite resection or allogeneic hematopoietic stem cell transplant. We screened drugs for those that prevent apoptosis of cells tested their effects an animal model disease.MethodsWe...
Background: Small randomized trials demonstrated that a lower compared with higher dialysate temperature reduced the average drop in intradialytic blood pressure. Some observational studies was associated risk of all-cause mortality and cardiovascular mortality. There is now need for large trial compares effect low vs high on major outcomes. Objective: The purpose this study to test outpatient hemodialysis centers (1) personalized temperature-reduced protocol or (2) standard-temperature 4...
Abstract Background Genetic defects of pediatric-onset inflammatory bowel disease (IBD) provide critical insights into molecular factors controlling intestinal homeostasis. NOX1 has been recently recognized as a major source reactive oxygen species (ROS) in human colonic epithelial cells. Here we assessed the functional consequences deficiency with respect to wound healing and migration by studying pediatric IBD patients presenting stop-gain mutation NOX1. Methods Functional characterization...
Major Outcomes with Personalized Dialysate TEMPerature (MyTEMP) is a 4-year cluster-randomized clinical trial comparing the effect of using personalized, temperature-reduced dialysate protocol versus temperature 36.5°C on cardiovascular-related death and hospitalization. Randomization was performed at level dialysis center ("the cluster").The objective to outline statistical analysis plan for MyTEMP trial.MyTEMP pragmatic, 2-arm, parallel-group, registry-based, open-label, trial.A total 84...
Dysfunction of visceral smooth muscle ("visceral myopathy") impairs bowel, bladder, and uterine function. Symptoms this life-threatening condition include massive intestinal distension with slow transit, vomiting, feeding intolerance, growth failure, poor bladder emptying, difficult vaginal delivery. The most common genetic cause myopathy is a heterozygous point mutation (R257C) in gamma actin (ACTG2). We genetically modified the WAe0009-A human embryonic stem cell line to carry c.769C>T...