Ionis Pharmaceuticals Inc. (($IONS)) announced an update on their ongoing clinical study. Ionis Pharmaceuticals Inc. is conducting a Phase 3 study ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy and safety of GTX-102 (apazunersen), an ...

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 ...

Colin Farrell has typically been very private about his home life, but now he’s opening up about his experience having a child with Angelman syndrome. Despite being relatively common, affecting 1 in ...

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

DENVER — Treatment with a novel therapeutic led to clinically significant improvement in cognition for young persons with Angelman syndrome, Kemi Olugemo, MD, FAAN, said in this Healio video at the ...

ELK GROVE - The world turned upside down for an Elk Grove family when their young daughter received a rare and life-changing diagnosis. On Thursday, International Angelman Day, they're shedding light ...

CINCINNATI (WKRC) - Kendall Millette is a 17-year-old living with Angelman syndrome. Now she is in need of a kidney to save her life. Kendall's mom Julie says, "She's always smiling and engaging and ...

Colin Farrell revealed that his 20-year-old son, James, has been living with a disorder known as Angelman Syndrome. While speaking with PEOPLE in a recent interview, the Saving Mr. Banks star, 48, ...

Actor Colin Farrell is known for being seen and heard on the big screen, but his next venture is about ensuring others, particularly those with intellectual disabilities, are treated with the same ...