We investigate essential functions of biological processes and fundamental mechanisms of diseases and disorders affecting children’s health. Our lab-based programs are organized into virtual scientific neighborhoods that promote collaborations.
Our work translates the discoveries of basic science into promising clinical applications. Multidisciplinary teams of researchers collaborate on clinical research on a range of specialties and conditions organized into program areas.
We drive the research on biological, psychological, social, behavioral, and environmental causes and influencers of common and prominent child health problems to generate evidence for clinical and public health interventions.
The Quantitative Science pillar provides cutting-edge statistical and analytical support and computational techniques to maximize research impact and improve the health outcomes of pediatric patients.
Our diverse team of researchers are influential leaders in investigating pediatric health issues and diseases, and include physicians, scientists, postdocs, coordinators, statisticians, data analysts, trainees, and others who routinely collaborate with medical centers, academic institutions, and community partners across the globe.
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This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and
The objective of this common multicenter protocol is to test the hypothesis that algorithmic tools using clinical Neonatal Intensive Care Unit (NICU) cardiorespiratory monitoring data
This is an intermediate-size expanded access protocol to provide ONC201 to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 through clinical trials.
This is an open label study of everolimus in children with recurrent or progressive low-grade glioma.
Primary Objective: To explore associations between biomarkers of atopic dermatitis (AD) and: - Disease state and time course of AD, - Disease state and
A randomized trial to determine whether simultaneous treatment with spectacles and patching has an equivalent VA outcome compared with sequential treatment, first with spectacles
This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and
This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or
This study is about an anticancer drug called ponatinib which is a tyrasine kinase inhibitor given with chemotherapy to children, teenagers, and young adults up to 21 years of age
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are
