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 therapy, tividenofusp alfa.

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 ...

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 ...

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 ...

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 ...

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

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 ...

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

GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on its LSD (lysosomal storage disorder) pipelines at the WORLD Symposium 2025, held ...