A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
To find out the effects of Maralixibat on the treatment of PFIC.
To find out the effects of Maralixibat on the treatment of PFIC.
Individuals with Down syndrome are at risk for the development of Alzheimer's disease. This study seeks to explore if neurodegenerative biomarkers of this condition are present in childhood. In addition, this study will explore the possibility of these same biomarkers being present in children with autism spectrum disorder and other neurodevelopmental conditions.
To test the effectiveness of an addiction-based weight loss intervention, embodied first as a smartphone app with telephone coaching (AppCoach) compared to (1) addiction model based weight-loss app alone (App) and (2) multi-disciplinary in-clinic weight loss intervention (Clinic) on weight outcomes of overweight and obese adolescents at 3, 6, 12 and 18 months post enrollment.
To evaluate long-term safety of Humira in pediatric patients (between the ages of 6 and 17 years inclusive at the time of enrollment) with moderately to severely active CD who are prescribed and treated in accordance with the approved local Humira product label under the conditions of routine clinical setting.
The objective of this study is to learn how anemia may affect oxygen delivery to the brain as measured by magnetic resonance imaging (MRI). By studying this, we hope to better identify people who may benefit from correction of their anemia.
An investigation of the utility of non-invasive transcranial Doppler (TCD) ultrasound in the prediction of moyamoya disease in persons with Down syndrome with the goal of identifying cerebrovascular disease before it causes a stroke.
The overall objective is to assess the influences that repeated head impact accelerative events (both impact and nonimpact) and concussions have on high school athletes as measured by brain structure and function, cognition, and behavior.
Comparing brain MRIs, blood brain barrier integrity and cognitive performance in those with and without Long COVID symptoms and in those that have and have notbeen previously hospitalized with COVID.
The purpose of the study is to learn about how to improve access to care during young adulthood and to assess if telehealth is an acceptable form of care.
This study will evaluate the effect of Denosumab on lumbar spine mineral density (BMD), as assessed by dual-energy X-Ray absorptiometry (DEXA) at 12 months in children 5 to 17 years of age with GIOP.