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

“Broken skin barriers” are real. They’re the subject of great interest from " skinfluencers " on TikTok, but they’re also ...

This work presents important findings regarding the interaction of the monkeypox virus (MPXV) attachment H3 protein with the cellular receptor heparan sulfate and the use of this information to ...

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

Denali Therapeutics公司今日公布了在研疗法tividenofusp alfa（DNL310），在47名亨特综合征（MPS ...

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

Denali Therapeutics (DNLI) announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome in the 24-week ...

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 ...

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 ...

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued ...

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). Per ...