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 29, 2024

Dubai Launches First Angelman Clinic in GCC

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

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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