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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November 6, 2024
Ionis Pharmaceuticals has released information regarding their clinical trial of ION582. The trial will move into Phase 3 and will be called Reveal. Ionis will be presenting more information at the FAST Global Science Summit on Saturday, November 9th […]
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August 12, 2024
Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). Highlights: Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from […]
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