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All of Us

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.

Burosumab

To collect information on the long-term effects of XLH on children and adults who have participated in burosumab clinical trials, adults and children who have not participated in burosumab clinical trials, and who may or may not have been treated with burosumab during the course of the study.

Concussion Study

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.

CP bone

To investigate bone health in cerebral palsy and the relationship between walking and bone health.

Diurnal Study

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.

HIGH DOSE ERYTHROPOIETIN FOR ASPHYXIA AND ENCEPHALOPATHY (HEAL)

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.