MIDDLETON, Mass., Nov. 6, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

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 ...

Angelman syndrome is a rare condition that causes problems with growth and development. It’s caused by a genetic mutation (a change in your genes) that affects the nervous system. It’s named after ...

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, ...

Broken Hill man Mitch McKenny has run more than 1,100 kilometres to Newcastle to raise awareness and funds for Angelman syndrome. Mr McKenny was inspired to perform the feat when the son of his close ...

CARLSBAD, Calif. - Ionis Pharmaceuticals (NASDAQ:IONS), whose stock has surged over 80% in the past six months and currently trades near its 52-week high of $62.08, announced Tuesday that its ...

CHESHIRE, Conn. (WTNH) — The General Motors charity car show was held on Sunday in Cheshire, benefitting families with children diagnosed with Angelman Syndrome. Quinnipiac students create customized ...

3-year-old Bellamy Bresaw of Bow was diagnosed with Angelman Syndrome at nine months old. Credit: Delores Flanders / Courtesy Delores Flanders and her family are hosting a fundraiser at Boutwell’s ...

MIDDLETON, Mass., May 12, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

Angelman syndrome is a rare genetic disorder that mainly affects the nervous system and causes developmental delays, speech difficulties, and movement problems. The condition has recently gained more ...

Irish actor Colin Farrell has movingly detailed the choice to place his 21-year-old son in long-term care. The Dublin native opened up on the journey that first saw his son James incorrectly diagnosed ...