Ultragenyx (RARE) Pharmaceutical announced that the first patient has been dosed in the Aurora study evaluating the efficacy and safety of GTX-102, an investigational antisense oligonucleotide for Angelman syndrome. Data from the Aurora study will expand the population of Angelman patients treated to include both younger and older patients and those with other non-deletion AS genotypes not included in the Phase 3 Aspire study. The fully enrolled Phase 3 Aspire study of GTX-102 is focused on patients aged 4 to 17 years with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
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