Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...

Denali announces encouraging additional longer-term data from the phase I/II Hunter Syndrome study of its investigational ...

Joseph Arboleda-Velasquez of Harvard Medical School praised the results. “Ye’s group has now provided compelling evidence showing increased binding affinity of ApoE3 Christchurch for tau and its ...

California, USA-based rare disease company Ultragenyx Pharmaceutical yesterday released new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically-significant ...

Although AAV uses the ubiquitously expressed cell surface heparan sulfate proteoglycan (HSPG) as a receptor, the transduction efficiency of AAV vectors varies greatly in different cells and ...

NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating ...

This innovative bonding technique imitates the natural situation of the natural antithrombotic effect of heparan sulfate, thus presenting a 'blood-friendly' surface. Excellent and pliable handling ...

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. At the end of the prepared ...

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

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

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