Charlie Nuck of Cincinnati has a 6-year-old niece named Briar. She has a rare, neurogenetic disorder called Angelman syndrome ...

An Ohio uncle is on a cross-country mission, riding through Northeast Ohio this weekend to find a cure for a rare syndrome.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising stocks according to Wall Street analysts. On June 27, Ultragenyx Pharmaceutical announced that the US FDA granted Breakthrough ...

The General Motors charity car show was held on Sunday in Cheshire, benefitting families with children diagnosed with Angelman Syndrome. Angelman Syndrome affects one in ...

Oak Hill Bio, a biotechnology company focused on developing life-changing therapies for people with rare diseases, today announced the publication in Nature Medicine of the results of the rugonersen ...

Angelman syndrome is a rare neurodevelopmental disorder arising primarily from loss of function of the maternally inherited UBE3A gene. Research has shown that UBE3A plays a crucial role in ...

Investing.com -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) stock gained 2.2% premarket after the company announced that its experimental treatment for Angelman syndrome received Breakthrough ...

In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: Angelman syndrome. Just before his son James’ 21st birthday, the actor ...

We are grateful to the ARIA initiative for its vision and generous support of this transformative work. It has the potential ...

Loss of this 2-Mb domain on the paternal or maternal allele results in two neurogenetic disorders, Prader-Willi syndrome (PWS) or Angelman syndrome (AS), respectively.