NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has ...

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 ...

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 ...

All told, 97% of people in the medium and high dose groups saw an improvement in overall symptoms measured using the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C ...

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

Ultragenyx Pharmaceuticals RARE announced that the FDA has granted the Breakthrough Therapy designation to its ...

Polio is in the news, with vaccination under threat and recent outbreaks. This makes Alan Marshall’s classic disability memoir more relevant than ever.

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 ...