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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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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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 […]
Read moreRoche has made the difficult decision not to move forward with a new clinical trial for rugonersen and has initiated the search for an external partner to take over the development. The conclusion of this trial undoubtedly brings feelings of […]
Read moreAURORA, IL – Angelman Syndrome Foundation (ASF) proudly announces the launch of the Jacob Pritzker Fellowship Program in honor of late board president Fred Pritzker. The Jacob Pritzker Fellowship Program was created to address the ever-increasing need for more physicians who […]
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