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Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

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.

Be sure to check clinical trials.gov for recruitment status and participation criteria.

Related

Ultragenyx Community Update October 2024
Ionis Pharmaceuticals releases information on their planned phase 3 clinical trial for Angelman syndrome
Watch the ASF | FAST Ultragenyx Community Webinar recording
Neuren Pharmaceuticals announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). 

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