Ultragenyx报告说,GTX-102针对天使综合症治疗的第1/2阶段取得了积极成果。 Ultragenyx reports positive Phase 1/2 results for GTX-102, targeting Angelman syndrome treatment.

Ultragenyx制药公司宣布GTX-102(一种旨在治疗Angelman综合症 -- -- 一种罕见的神经基因紊乱症 -- -- 的抗血清寡核分裂化物)第1/2阶段有希望的结果。 Ultragenyx Pharmaceutical announced promising Phase 1/2 results for GTX-102, an antisense oligonucleotide aimed at treating Angelman syndrome, a rare neurogenetic disorder. 数据表明在关键认知领域有所改进,支持即将进行的第三阶段研究,该研究计划招收约120名病人。 Data indicate improvements in key cognitive areas, supporting the upcoming Phase 3 Aspire study, which plans to enroll around 120 patients. 该研究将侧重于认知增强,并收到了监管机构为其潜在益处作出的多次指定。 The study will focus on cognitive enhancements and has received multiple designations from regulatory bodies for its potential benefits.