Onlymyhealth19d
What Is Sanfilippo Syndrome: Symptoms, Treatment, and the Impact on Children’s Lives
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.
Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...
Ultragenyx reports Q4 EPS ($1.39), consensus ($1.27)
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
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 ...
Nasdaq8d
RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
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).