For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).

Am Freitag bestätigte Goldman Sachs seine Kaufempfehlung für die Aktien von Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) und hielt am Kursziel von 78,00 US-Dollar fest. Der Analyst der Investmentbank ...

GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on its ...

Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, stated, "The data support UX111's potential to provide meaningful benefits to children with Sanfilippo Syndrome, especially for those in ...

Discover how Ultragenyx achieved 29% revenue growth in 2024, set 2025 guidance at $640M-$670M, and advanced clinical progress toward profitability by 2027.

Ultragenyx ist auf die Entwicklung von Therapien für seltene und ultra-seltene genetische Erkrankungen spezialisiert und verfügt über ein Portfolio zugelassener Medikamente und Kandidaten in der ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Emil Kakkis; President, Chief Executive Officer, Director; Ultragenyx Pharmaceutical Inc Erik Harris; Executive Vice President, Chief Commercial Officer; Ultragenyx Pharmaceutical Inc Howard Horn; ...