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., June 27 ...

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 ...

Ultragenyx Pharmaceutical received breakthrough therapy designation from the U.S. Food and Drug Administration for its drug GTX-102, which is designed to treat people with Angelman syndrome. Angelman ...

Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome ...

In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: ...

Held at the Crystal Ballroom of the Millennium Biltmore Hotel in downtown Los Angeles, the Southern California Journalism Awards drew more than 2,500 entries, breaking last year’s record ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. The initial ...

Coach Aaron Clark and his wife, Darlene, who is battling cancer, found a second family on the field thanks to their flag ...

Angelman syndrome is a rare condition that causes problems with growth and development. It’s caused by a genetic mutation (a change in your genes) that affects the nervous system. It’s named ...

More information: Jacqueline Fátima Martins de Almeida et al, Molecular aspects of Angelman Syndrome: Defining the new path forward, Biomolecules and Biomedicine (2025). DOI: 10.17305/bb.2025.11724 ...

According to DelveInsight's estimates, the Angelman syndrome market size in the 7MM is expected to show positive growth during the forecast period (2025–2034), primarily driven by the increasing ...

Angelman Syndrome Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Angelman syndrome epidemiology in the 7MM, i.e., the United ...