This is a randomized clinical trial of epiphysiodesis techniques: percutaneous transphyseal screw epiphysiodesis versus percutaneous drill epiphysiodesis for the correction of leg length discrepancy....
This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN).
Central line-associated bloodstream infections (CLABSIs) are the most common healthcare-associated infection in children and are associated with morbidity and mortality. This study will attempt to...
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 natural history of...
The ultimate goal of this project is to develop methods that allow informed decision-making on the delivery time of fetuses that are at increased risk of stillbirth due to IUGR. In placenta related...
This study will combine three drugs: sorafenib, cyclophosphamide and topotecan.
Adding sorafenib to cyclophosphamide and topotecan may increase the effectiveness of this combination. The...
Recently, researchers and clinicians have examined many different forms of concussion testing aimed to assess if a brain injury has occurred and to what degree it affects the individual being tested....
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the...
Neonates, children with single ventricle congenital heart disease, and those undergoing multiple complex cardiac surgeries are at high risk of increased perioperative blood loss, and blood product...
Continuation of the CARRA Registry as described in the protocol will support data collection on patients with pediatric-onset rheumatic diseases. The CARRA Registry will form the basis for future...
The Genomic Sequencing for Childhood Risk and Newborn Illness (the BabySeq Project) is a research study exploring the use of genomic sequencing in newborns. The National Institutes of Health is...
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
Background:
- Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. The NIH helped create a network of medical...
The purpose of this study is to comprehensively characterize PMS using standardized medical, cognitive, and behavioral measures and to track the natural history of the syndrome using repeated...
The purpose of this study is to determine cross-sectional and longitudinal medical, behavioral, and cognitive differences between PTEN ASD and other groups, as well as to identify cognitive, neural...
The purpose of this study is to characterize the developmental phenotype of ASD and ID and to identify biomarkers using advanced MRI methodology and electrophysiological biomarkers of synaptic...
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations...
