For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman syndrome. James was just a toddler when he was diagnosed with the rare ...

An Ionis Pharmaceuticals drug in development for Angelman syndrome has mid-stage clinical trial results showing improvement across a range of measures of this rare neurodevelopmental disorder, and the ...

In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as ...

Angelman syndrome is a rare disease that disrupts the normal development and function of cells, particularly in the nervous ...

Rebecca Burdine knew something was wrong the day her baby girl was born. Sophie was born without a natural sucking reflex, making it nearly impossible to eat. Later, it became clear that she had ...

Colin Farrell is opening up about his son James' Angelman syndrome diagnosis and what he's doing to help others with intellectual disabilities. "I want the world to be kind to James," Farrell told ...

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 ...

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, ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. The initial participant in the pivotal Phase 3 REVEAL trial, which is assessing ION582 ...

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 ...

This post was updated on April 24, 2019 to incorporate feedback from parents. Names have been changed to protect anonymity. Is this child happy? Source: Joel Frohlich (AI generated with Midjourney) ...