May 12, 2025
MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for MVX-220, an investigational […]
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April 15, 2025
In June 2023, Roche announced they would not be moving forward with a clinical trial for rugonersen, an investigational ASO for AS, and would be looking for an external partner to take over the development. Oak Hill Bio has entered […]
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February 10, 2025
Introducing the Angelman Syndrome Global Community Advisory Board (CAB) As we prepare for International Angelman Day, we are thrilled to introduce the Angelman Syndrome Global Community Advisory Board (CAB)! This remarkable group of parent advocates and expert parents/carers has come […]
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December 19, 2024
Ultragenyx Pharmaceutical Inc. announced today that the first patient(s) has/have been dosed in the pivotal Phase 3 Aspire study evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. Check clinicaltrials.gov for recruitment status and participation criteria. […]
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