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 randomized phase III trial compares how well combination chemotherapy works when given with or without bortezomib in treating patients with newly diagnosed T-cell acute
This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study will use a new treatment approach based on each patient's tumor gene
This phase III trial studies the side effects and how well risk-based therapy works in treating younger patients with newly diagnosed liver cancer. Surgery, chemotherapy drugs
Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. The purpose of this study is to collect the pertinent clinical
This is a multicenter, safety and pharmacokinetic trial to determine the MTD and/or select a recommended phase 2 dose (RP2D) of vemurafenib in children with recurrent or refractory
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the
The purpose of this study is to determine whether Heat Shock Protein Peptide Complex-96 (HSPPC-96) Vaccine is an feasible and safe treatment for pediatric patients with
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are heterozygous for F508del and a
The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular
The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a
