Angelman syndrome is a rare neurodevelopmental disorder arising primarily from loss of function of the maternally inherited UBE3A gene. Research has shown that UBE3A plays a crucial role in neuronal ...

Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 “The accelerated ...

Ultragenyx Pharmaceutical has dosed the first participant in Aurora, a study evaluating GTX-102 (apazunersen) for angelman syndrome (AS). The rare, neurogenetic condition results from the loss of ...

ORLANDO, Fla.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that ...

NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) ...

Neurons with a fluorescent reporter for paternal UBE3A gene activity show only a background low fluorescent glow when treated with a vehicle control, but show a bright fluorescent glow when treated ...

Positive End of Phase 2 discussion with FDA, including alignment on Phase 3 design Bayley-4 expressive communication selected as Phase 3 study primary endpoint Initiation of ION582 Phase 3 study ...

Add Yahoo as a preferred source to see more of our stories on Google. HOLLYWOOD, CALIFORNIA - MARCH 12: Colin Farrell attends the 95th Annual Academy Awards on March 12, 2023 in Hollywood, California.

Niigata, Japan - Researchers from Brain Research Institute, Niigata University, Japan have revealed that environment influences social behaviours in autism. By using zebrafish that have a mutation in ...

Researchers at Beth Israel Deaconess Medical Center (BIDMC) have gained new insight into the genetic and neuronal circuit mechanisms that may contribute to impaired sociability in some forms of Autism ...