04:25 PM EST, 12/19/2024 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said Thursday the first patient has been dosed in the phase 3 study of its drug candidate GTX-102 for the treatment of Angelman syndrome, a rare neurodegenerative disorder.

The company said it intends to enroll about 120 children in the Aspire study to evaluate the efficacy and safety of GTX-102.

Ultragenyx also said it is on track to start the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025.