April 15, 2025

Oak Hill Bio Obtain Global Rights for Rugonersen

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

Community Advisory Board

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

First Patient(s) Dosed in Ultragenyx Phase 3 Aspire Study

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 Releases News about Clinical Trial of ION582

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 Releases Results from Phase 2 Clinical Trial of NNZ-2591

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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July 17, 2024

Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA

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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