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Abiraterone Acetate in Children with Classic 21-Hydroxylase Deficiency

The purpose of this study is to (1) determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels in prepubertal children with CAH secondary to 21-hydroxylase deficiency (Phase 1 trial), and (2) assess the utility of abiraterone acetate in prepubertal children with CAH as adjunctive therapy to minimize excessive androgen secretion and allow more physiological glucocorticoid replacement (Phase 2 trial).

AD Biomarkers Study

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.

Adrenas CAH (Congenital Adrenal Hyperplasia) Study

This is a Phase 1/2, first-in-human, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of BBP-631 administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia (CAH) (simple virilizing or salt-wasting, Group 1) or with classic salt-wasting CAH (Group 2) due to 21-hydroxylase deficiency (21-OHD) and who are monitored for 24 weeks post-treatment. All participants who receive BBP-631 will be followed for an additional 4.5 years for safety and efficacy in a separate long term follow up protocol (Study CAH-399). In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

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.

Bioflex Sensor

The purpose of this study is to evaluate a sensor that transmits intracranial pressure, cerebrospinal fluid flow, and makes sure the path of the catheter remains open.

Brain Blood Flow

This study uses sensitive measures of brain blood flow an its response to change to inhaled oxygen levels to determine how the brain protects its oxygen supply.

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.