Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in ...
The BEAM study is a multicenter, prospective, observational study in children supported on extracorporeal membrane oxygenation (ECMO). The primary goals of this study are to develop and refine a brain ...
Cervical spine injuries (CSI) are serious, but rare events in children. Spinal precautions (rigid cervical collar and immobilization on a longboard) in the prehospital setting may be beneficial for ...
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants. This ...
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 ...