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.
The biotechnology company, Ultragenyx, has reported a successful fourth quarter and full year 2024 financial results ...
Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
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).
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