Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A ...

Bike 4 Briar had made it’s way to Fulton, NY, where they were welcomed at City Hall . Lunch was provided and a snack donation was made for their journey.  Bike 4 Briar is a ...

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 ...

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 ...

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.

Clark Olofsson, the notorious Swedish criminal who inspired the popular psychological term, died after a long illness, his family announced.