International Angelman Day also marks the birthday of Dr. Harry Angelman, the British pediatrician who first identified the ...

More than tequila: the untold story of love, resilience, and purpose fueling the rise of Señor Rio in Los Angeles. When ...

Positive interim efficacy data in 2025 underscore potential of ETX101 as a one-time, disease-modifying medicine for Dravet syndrome –– Internal GMP manufacturing capable of supporting pivotal trial ...

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced that Kartik Ramamoorthi, Ph.D., Chief Executive ...

She shocked doctors by living to 41 with rare ‘butterfly’ disorder. She also picked up a celebrity friend along the way - IN FOCUS: EXCLUSIVE: ‘We sat down beside each other, and we just felt like ...

As it kicks off a pivotal phase 3 trial of its investigational treatment for Angelman syndrome (AS), Ionis Pharmaceuticals is laying the groundwork for a potential launch. After spending the last ...

BATON ROUGE, La. – LSU women’s basketball will help raise awareness and money for Turner Syndrome, a cause close to Head ...

In Yakima, four-year-old Kennedy, a Miracle Child, shows incredible spirit overcoming Angelman Syndrome with the help of ...

People who struggle with imposter syndrome believe that they are undeserving of their achievements and the high esteem in which they are, in fact, generally held. They feel that they aren’t as ...

“There’s this old thought that somebody with Down syndrome won’t be able to get a job or live independently,” says Kishore Vellody, MD, a professor of pediatrics at the University of Pittsburgh School ...