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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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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Ben Philpot, PhD and his lab at UNC Chapel Hill have identified a small molecule that could be a potential gene therapy treatment for AS. The ASF first funded Dr. Philpot’s pilot study to partner with Pfizer to evaluate over […]
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