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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The purpose of this research study is to learn if the study drug RAD001 can shrink or slow the growth of low-grade gliomas in children with Neurofibromatosis type 1 (NF1).
This trial examined the outcome benefit to patients of adding a new chemotherapy drug combination to the established treatment approach for patients with extracranial Ewing
Atopic dermatitis (AD), also known as eczema, is the most common inflammatory skin disorder of children, affecting 10-20% of children and 1-2% of adults. This skin disorder can
Dolutegravir (DTG) is an HIV drug in the integrase inhibitor drug class. This study evaluated the pharmacokinetics (PK), safety, tolerability of and immune response to DTG when used
Treatment for pediatric acute myeloid leukemia (AML) involves intensive chemotherapy regimens that result in periods of profound neutropenia leaving patients susceptible to severe
The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A
This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors,
This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT)
The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will
A randomized, blinded, controlled trial to evaluate growth velocity and clinical outcomes in infants with single ventricle physiology fed an exclusive human milk diet prior to, and
A total of 1900 pregnant women in the 1st trimester (633 women living with HIV (WLHIV) initiating Dolutegravir (DTG)-based antiretroviral therapy (ART) in pregnancy, 633 WLHIV
