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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Study to evaluate the safety of ridinilazole in adolescent subjects and how ridinilazole is metabolized.
This study evaluates the performance of a non-invasive thermal measurement device for assessing CSF shunt flow. Patients with an existing implanted shunt and symptoms of shunt
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease
This phase III trial compares standard chemotherapy to therapy with liposome-encapsulated daunorubicin-cytarabine (CPX-351) and/or gilteritinib for patients with newly diagnosed acute
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and
The goal of this study is to evaluate the safety of the study drug PTC596 (Unesbulin) taken in combination with radiotherapy (RT) when given to pediatric patients newly diagnosed with
Severe pediatric acute respiratory distress syndrome (PARDS) is a life-threatening and frequent problem experienced by thousands of children each year. Little evidence supports
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN
SMARTT will estimate the incidence of conditions and diagnoses potentially related to in utero exposure to antiretroviral therapy and/or exposure in the first two months of life
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II BDCPA operation and to
The primary purpose of this study is to evaluate the efficacy of alogliptin 25 milligram (mg) once daily compared to placebo when administered as monotherapy, or when added onto a
