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Your search keyword '"Vincenzi, Brenda"' showing total 1 results
Start Over Author "Vincenzi, Brenda" Publisher escholarship, university of california
1 results on '"Vincenzi, Brenda"'

1. Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS).

Author

Tjeertes, Jorrit, Bacino, Carlos, Bichell, Terry, Bustamante, Mariana, Crean, Rebecca, Jeste, Shafali, Komorowski, Robert, Krishnan, Michelle, Miller, Meghan, Nobbs, David, Ochoa-Lubinoff, Cesar, Parkerson, Kimberly, Rotenberg, Alexander, Sadhwani, Anjali, Shen, Mark, Squassante, Lisa, Tan, Wen-Hann, Vincenzi, Brenda, Wheeler, Anne, Hipp, Joerg, Berry-Kravis, Elizabeth, and Bird, Lynne

Subjects
Angelman syndrome, Clinical outcome assessments, Clinical trials, Digital health technology, EEG, Endpoint development, Natural history, Sleep, UBE3A, Humans, Angelman Syndrome, Prospective Studies, Pandemics, COVID-19, Electroencephalography
Abstract

BACKGROUND: Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by the absence of a functional UBE3A gene, which causes developmental, behavioral, and medical challenges. While currently untreatable, comprehensive data could help identify appropriate endpoints assessing meaningful improvements in clinical trials. Herein are reported the results from the FREESIAS study assessing the feasibility and utility of in-clinic and at-home measures of key AS symptoms. METHODS: Fifty-five individuals with AS (aged

Published
2023

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