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.
The goal of this study is to help researchers across the nation develop and tailor healthcare treatments to the individual. They will do this by collecting data & biospecimens that may enable a broad spectrum of research studies.
This prospective, longitudinal study aims to measure and describe the amount and frequency of physical activity, feeding, and sleeping across days and months in infancy.
We want to learn more about how cancer treatment, such as immunotherapy (i.e., CAR T cell treatment), affects the brain, learning, and behavior in patients with Acute Lymphoblastic Leukemia (ALL).
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.
To investigate bone health in cerebral palsy and the relationship between walking and bone health.
This study is a randomized, double-blind, active-controlled, titrated, parallel arm, multicenter study. It will compare the efficacy, safety and tolerability of twice daily Chronocort with twice daily IRHC (Cortef®) over a randomized treatment period of up to 52 weeks in participants aged 16 years and over with known classic CAH due to 21-hydroxylase deficiency. The primary efficacy assessment of biochemical responder rate and the key secondary assessments of dose responder rate and mean total daily dose will be assessed after 52 weeks of randomized treatment.
We would like to study how the body responds to the flu infections or flu vaccine in mothers and their children.
The first Phase IIb clinical trial in Down syndrome regression disorder (DSRD). An open-label, randomized study comparing the safety and efficacy of lorazepam, intravenous immunoglobulin (IVIg) and the Janus Kinase inhibitor (JAK inhibitor), tofacitinib.
1) To determine if 5 doses of Epo (Erythropoietin) 1000 U/kg (birth weight) intravenous (IV) reduces the rate of death or neurodevelopmental impairment (mild, moderate, or severe) at 24 months of age.
2) To assess safety of Epo.
3) To determine whether Epo decreases the severity of HIE-induced brain injury as evidenced by early MRI and plasma biomarkers of brain injury.
