December 19, 2024
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 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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