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.

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 ...

Nancy Brady has been gratified to see the tool she and colleagues pioneered over a decade ago to measure the growth of infants' pre-speech communication skills translated into several languages and ...

Massachusetts General Hospital in Boston announced the opening of its new Angelman Syndrome Clinic — one of only two in the country, according to the release. The clinic was established through a ...

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

NOVATO, Calif., Oct. 30, 2025 (GLOBE NEWSWIRE) -- (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy ...