Researchers from the University of Tokyo reveal how the presynaptic Ube3a E3 ligase, a causal factor in Angelman syndrome, eliminates neural chat. The study helps find a better drug target for the ...
Colin Farrell says his personal life has inspired him to launch a new foundation aimed at helping people with intellectual disabilities. The actor, 48, recently launched the Colin Farrell Foundation, ...
The Pinball Lounge at 78th Street Sudios offers an ideal setting for this unique fundraiser. The event runs from 4-6 pm, providing families with an accessible Sunday afternoon activity. True to its ...
This post was updated on April 24, 2019 to incorporate feedback from parents. Names have been changed to protect anonymity. Is this child happy? Source: Joel Frohlich (AI generated with Midjourney) ...
NOVATO, Calif., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious ...
Ultragenyx Pharmaceutical Inc. announced the dosing of the first patient in its Phase 3 Aspire study, which is investigating the safety and efficacy of GTX-102, an investigational antisense ...
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 ...
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) ...
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