Unrecognized coarctation of the aorta (CoA) is a life-threatening component of congenital heart disease (CHD) in which narrowing of the aorta causes obstructed blood flow to the lower half of the body ...
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo ...
The goal of the study is to examine multiple markers of anthropometrics, body composition, sarcopenia and frailty and compare them to dual energy X-ray absorptiometry (DXA) output, which is considered ...
Patent Ductus Arteriosus is a developmental condition commonly observed among preterm infants. It is a condition where the opening between the two major blood vessels leading from the heart fail to ...
This is a multi-center, double-blind, placebo-controlled, randomized trial of dupilumab adjunctive therapy for prevention of asthma exacerbations in urban children and adolescents with T2-high ...
Multi-system Inflammatory Syndrome in Children (MIS-C) is a new condition related to COVID-19, the study investigators are still learning about its causes, effects, and long-term impact. "Long-Term ...
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC ...
Randomized Controlled Trial comparing a coach-enhanced digital cognitive behavioral intervention (d-CBI) (RxWell) + treatment as usual (TAU) versus TAU alone for moderate depression as determined by ...
This is a multi-center, longitudinal study which will characterize the gene expression profiles and transcriptomic endotypes that underlie mild and moderate-severe Atopic dermatitis (AD) and will ...
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.