Fierce Biotech

Ultragenyx fails phase 3 Angelman test, plots ‘significant expense reductions’

Ultragenyx had been flying high after its recent FDA approval for Genglycos—but the company has been brought back down to ...
Stocktwits on MSN

RARE stock heads for record low: Angelman trial miss leads to Wall Street downgrades – this analyst sees another problem ahead

Ultragenyx’s GTX-102 failed to meet its main goal of improving cognitive function in children aged 4 to 17. ・William Blair does not expect any major event over the next 12 months that could ...
Yahoo Finance

MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome

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 ...
Yahoo

What is Angelman syndrome? The rare genetic condition affecting Colin Farrell’s son

Actor Colin Farrell has opened up about the difficult decision to move his son James, who has Angelman syndrome, into a long-term care facility. In an interview this week the actor, 48, revealed he ...
Medical Xpress

Molecular strategies for Angelman syndrome explored in study

A new review sheds light on the complex molecular mechanisms behind Angelman syndrome (AS), a rare neurogenetic disorder, and explores how cutting-edge gene-targeting therapies may offer more ...