Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome
Ionis Pharmaceuticals issued a press release today regarding their Phase 1/2a HALOS clinical trial of ION582 for individuals living with Angelman syndrome. Join the livestream on July 24th starting at 3:10pm ET where Ionis and other industry leaders will present information to parents and caregivers at the Angelman Syndrome Foundation (ASF) Family Conference. You can […]
Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program
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 here.
Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome
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, which will be streamed for those who cannot attend. Read the community letter from Ionis […]
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
We’re thrilled to share an update from Ultragenyx regarding 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 interim data demonstrates that treatment with GTX-102 resulted in rapid, multi-domain improvements that continued during […]
Ultragenyx Pharmaceutical Ultragenyx announces completion of enrollment in Global Phase 1/2 Trial of GTX-102
Ultragenyx announced the completion of enrollment in Global Phase 1/2 Trial of GTX-102 in pediatric patients living with Angelman syndrome and data from at least 20 patients enrolled in dose expansion cohorts anticipated in the first half of 2024. Read press release here.
An update on Roche’s trials of rugonersen (RO7248824) in Angelman syndrome
We are sad to report that Roche has decided they will not initiate any new trials of rugonersen (RO7248824) in Angelman syndrome. If you are currently participating in the study, you should be hearing from the Investigator or their team at the site where your child is enrolled regarding important information about next steps for your […]