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.
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