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RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for ...
Ultragenyx announces new data on UX111 AV gene therapy
Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...
The Pharma Letter16h
Ultragenyx updates on UX111 AAV gene therapy
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 ...
FierceBiotech12h
Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...