Article

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

2023-01-24

Abstract excerpt

<title>Abstract</title> <p>Background: Angelman syndrome (AS) is a rare neurodevelopmental disorder with developmental, behavioral, and medical challenges caused by absence of functional UBE3A. AS has unmet treatment needs and no approved therapies address its underlying pathophysiology. To develop new therapies, comprehensive data are needed for identification of appropriate endpoints to assess meaningful impro...

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Literature Corpus work: 3a77a9f5-f88b-5827-a4a7-ab542060db6d
DOI: 10.21203/rs.3.rs-2484857/v1

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