Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome [Yahoo! Finance]
Ultragenyx Pharmaceutical Inc. (RARE)
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Source: Yahoo! Finance
The primary endpoint of the study is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint is the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Was this helpful? Yes No What is the current status of treatment options for Angelman syndrome? There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement of symptoms can potentially be achieved at any age. Was this helpful? Yes No What is Angelman syndrome and how does it affect individuals? Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. It affects cognitive and motor function, making walking, communicating, and performing many everyday tasks more difficult for indi
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