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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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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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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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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Ionis Pharmaceuticals issued a press release today regarding their Phase 1/2a HALOS clinical trial of ION582 for individuals living with Angelman syndrome. Read the Ionis press release here. Livestream Update on July 24 Join the livestream on July 24th at […]
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Today, Ultragenyx Pharmaceuticals shared a press release announcing a “Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program.” Read the full press release. Livestream Update on Wednesday, July 24 Tune into the LIVESTREAM updates at the ASF Family Conference on […]
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Today the Canadian Angelman Syndrome Society (CASS) and the Angelman Syndrome Foundation (ASF) announced that the two charities will collaborate with a shared goal of increasing research and support to Angelman syndrome (AS) families. United, the organizations will have the […]
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Angelman Syndrome Foundation and Dup15q Alliance Announce Development of the International Clinical Research Network in efforts to help those living with 15q syndromes. Thousands of families affected by Angelman syndrome (AS) and dup15q syndrome will soon have greater access to […]
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