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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May 16, 2024

Ionis Announces Positive Results from Halos Phase 1/2a

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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