New Collaboration Aims to Accelerate Angelman Syndrome Study Data Analysis
A new collaboration is underway to accelerate data analysis and publication from an ongoing and a previous natural history study on Angelman syndrome. The study, expected to conclude in August 2022, will analyze data from potentially over 450 patients to identify relevant goals for clinical trials.
The ongoing study, funded by the U.S. Food and Drug Administration and currently recruiting 150 patients, aims to better understand disease symptoms and develop more sensitive and specific measures for clinical trials. It follows a previous study funded by the National Institutes of Health, which ran from 2006 to 2014 and had over 300 participants.
The collaboration involves the Angelman Syndrome Biomarker and Outcome Measure (ABOM) Consortium, the Research Triangle Institute International (RTI), and Boston Children's Hospital. Experts supporting the analysis include clinical geneticists, neurologists, and research scientists specializing in neurogenetic disorders.
The goal of this collaboration is to better understand the common features of Angelman syndrome over time, including key clinical characteristics, medical complications, and quality of life. The Foundation for Angelman Syndrome Therapeutics (FAST) is supporting this research partnership to speed up data analysis and publication.
