The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels ...
Creation of a pediatric robotic spine surgery registry will allow for data collection and analysis on the coupled use of robotics and navigation, as well as patient-specific rods in pediatric spine ...
The mission of the SARP is to improve the understanding of severe asthma through integrated study of its clinical and biological features and to evaluate their changes over time. The ultimate goal of ...
Comparison of casting and bracing for the treatment of idiopathic early onset scoliosis
The purpose of this study is to test a pediatric narcolepsy patient reported outcomes tool to assess pediatric narcolepsy symptoms and their effect on daily functioning and quality of life. The goal ...
The investigators have designed a 2-center, pilot feasibility, randomized controlled trial (PROXIMUS) to determine the feasibility and safety of a larger multi center, randomized open-label trial ...
This is a multicenter longitudinal study that aims to validate a set of measures that were previously identified as promising candidate biomarkers and/or sensitive and reliable objective measures of ...
The objective of this postmarket, retrospective, single center study is to evaluate the clinical outcomes of patients who have received PhotoFix® Decellularized Bovine Pericardium (PhotoFix) as a ...
The study objective is to compare neurodevelopmental (ND) and behavioral outcomes between children with Down syndrome (DS) who had complete atrioventricular septal defect (CAVSD) repair and children ...
This is a multi-center, non-interventional registry to create and maintain a database of participants to serve as a recruitment source for current and future DAIT NIAID-sponsored Childhood Asthma in ...