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 ...

Nuck said Angelman syndrome research and clinical trials are very promising, but do not receive much funding because so few people know about the condition. Therefore, Bike for Briar’s main goal ...

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 ...

NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug ...

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 ...

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 ...

Company News Published 06/27/2025, 08:09 AM 0 FDA grants breakthrough therapy status to Ultragenyx’s Angelman drug RARE 1.14% ...

Ultragenyx Pharmaceuticals' GTX-102 earns FDA Breakthrough status after early data shows sustained progress in Angelman syndrome patients.

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 (TADAWUL: 2070) Inc. (NASDAQ: RARE) stock gained 2.2% premarket after the company announced that its experimental treatment for Angelman syndrome received ...