Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif ...

The Aurora study will evaluate GTX-102 across other Angelman syndrome genotypes and ages and is expected to initiate in the second half of 2025.

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

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 ...

Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in ...

Bike 4 Briar cyclists cross the country to support Angelman syndrome research The Bike for Briar team, consisting of Todd Downey and Charlie Nuck, is biking 4,200 miles across the U.S. and passed ...

On June 27, Ultragenyx Pharmaceutical announced that the US FDA granted Breakthrough Therapy Designation for GTX-102 (apazunersen) as a treatment for Angelman syndrome.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising stocks according to Wall Street analysts.

An Ohio uncle on a cross-country mission will be riding through Northeast Ohio this weekend. Charlie Nuck of Cincinnati has a 6-year-old niece named Briar. She has a rare, neurogenetic disorder called ...