Ultragenyx Pharmaceutical will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation Family Conference and Research Symposium in Sandusky, Ohio. The ASF data presentation will review the previously disclosed Phase 1/2 results presented at AAN and an update on plans to initiate a Phase 3 pivotal trial by the end of the year.
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