Ultragenyx preps for pivotal trial in Angelman syndrome, an area where others fell short: #AAN24

Ul­tragenyx is inch­ing clos­er to a Phase 3 study for its An­gel­man syn­drome pro­gram, known as GTX-102.

In a Phase 1/2 up­date Mon­day morn­ing ahead of its Tues­day night pre­sen­ta­tion at the an­nu­al Amer­i­can Acad­e­my of Neu­rol­o­gy meet­ing, Ul­tragenyx was light on da­ta specifics but said a piv­otal tri­al is ex­pect­ed to launch some­time this year. Its stock price $RARE fell by as much as 14% pre­mar­ket but was down about 9% in ear­ly Mon­day trad­ing.

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