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IN THE NEWS

Latest press releases, articles and announcements

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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