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.
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 primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized
To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular
Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001.
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy. The study consists of a
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 main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil
