Angelman syndrome is a rare disease that disrupts the normal development and function of cells, particularly in the nervous system, leading to severe developmental delays and learning disabilities.

Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, ...

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 ...

Mitch McKenny is unlikely to run again after spending four weeks running from Broken Hill to Newcastle, including the last ...

MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the first patient has been dosed in ...

Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On October 30, 2025, Ultragenyx Pharmaceutical announced the dosing of the first ...

SARASOTA, Fla. and NOVATO, Calif., Sept. 27, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of GTX-102, ...

Ionis Pharmaceuticals IONS announced that the FDA has granted the Breakthrough Therapy designation to its pipeline candidate, ION582, for the treatment of Angelman syndrome (AS), a rare neurological ...