Actor Colin Farrell recently disclosed that his son, James, has Angelman syndrome, one of the driving factors behind Farrell's support for finding a cure for Angelman syndrome and for launching the ...
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 ...
NOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps ...
NOVATO, Calif., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the ...
MIDDLETON, Mass., Sept. 22, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...
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 ...
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 NOVATO, Calif., ...
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 ...
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