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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July 22, 2024
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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July 17, 2024
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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July 8, 2024
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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May 29, 2024
Dubai Health, the first integrated academic health system in Dubai, has partnered with the Angelman Syndrome Foundation to establish the GCC’s first dedicated clinic at Al Jalila Children’s Hospital. This is the first-of-its-kind clinic in the region and aims to […]
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May 16, 2024
Exciting news for the Angelman syndrome community! Ionis’ Angelman syndrome program, ION582, has just shared positive preliminary results from the Phase 1/2a HALOS clinical trial. Ionis will be providing an update at the upcoming Angelman Syndrome Foundation meeting in July. […]
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April 15, 2024
See a letter to the Angelman community. Ultragenyx reported positive interim data from the ongoing Phase 1/2 study in patients living with deletion genotype Angelman syndrome after treatment with investigational GTX-102. From company press release: “The totality of these […]
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